79. 家族性高コレステロール血症（ホモ接合体） Homozygous familial hypercholesterolemia Clinical trials / Disease details
臨床試験数 : 145 / 薬物数 : 114 - (DrugBank : 26) / 標的遺伝子数 : 8 - 標的パスウェイ数 : 17
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05611528 (ClinicalTrials.gov) | February 21, 2023 | 31/10/2022 | Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia | Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia in a Real Life Setting in Canada | Homozygous Familial Hypercholesterolemia | Drug: Evinacumab | Daniel Gaudet | Ultragenyx Pharmaceutical Inc | Recruiting | 18 Years | N/A | All | 10 | Phase 3 | Canada |
| 2 | NCT05217667 (ClinicalTrials.gov) | April 22, 2022 | 20/1/2022 | Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH) | Phase 2 Study to Evaluate the Safety and Efficacy of ARO-ANG3 in Subjects With Homozygous Familial Hypercholesterolemia (HOFH) | Homozygous Familial Hypercholesterolemia | Drug: ARO-ANG 3 Injection | Arrowhead Pharmaceuticals | NULL | Active, not recruiting | 16 Years | N/A | All | 18 | Phase 2 | United States;Australia;Canada;South Africa |
| 3 | EUCTR2020-004394-49-ES (EUCTR) | 31/03/2022 | 02/02/2022 | Study to Evaluate the Long-Term Efficacy and Safety of the Investigational Drug LIB003 for the Reduction of Cholesterol in Patients with Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease. | Open-Label Extension Phase 3 Study to Evaluate the Long-Term Efficacy and Safety of LIB003 in Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease, on Stable Lipid-Lowering Therapy Requiring Additional Low-Density Lipoprotein Cholesterol Reduction (LIBerate-OLE) - LIBerate-OLE | Patients with elevated LDL-C, with homozygous familial hypercholesterolemia (HoFH) and heterozygous familialhypercholesterolemia (HeFH), cardiovascular disease, or at high risk for cardiovascular disease. MedDRA version: 20.0;Level: LLT;Classification code 10057079;Term: Heterozygous familial hypercholesterolemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10007648;Term: Cardiovascular disease, unspecified;System Organ Class: 100000004849;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: LIB003 Product Code: lerodalcibep INN or Proposed INN: Lerodalcibep Other descriptive name: LIB003 | LIB Therapeutics, LLC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 2000 | Phase 3 | United States;France;Canada;Spain;Turkey;South Africa;Israel;Norway;Germany;United Kingdom;New Zealand;India | ||
| 4 | EUCTR2020-004394-49-NO (EUCTR) | 12/08/2021 | 25/05/2021 | Study to Evaluate the Long-Term Efficacy and Safety of the Investigational Drug LIB003 for the Reduction of Cholesterol in Patients with Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease. | Open-Label Extension Phase 3 Study to Evaluate the Long-Term Efficacy and Safety of LIB003 in Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease, on Stable Lipid-Lowering Therapy Requiring Additional Low-Density Lipoprotein Cholesterol Reduction (LIBerate-OLE) - LIBerate-OLE | Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease MedDRA version: 20.0;Level: LLT;Classification code 10057079;Term: Heterozygous familial hypercholesterolemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10007648;Term: Cardiovascular disease, unspecified;System Organ Class: 100000004849;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: LIB003 Product Code: lerodalcibep INN or Proposed INN: Lerodalcibep Other descriptive name: LIB003 | LIB Therapeutics, LLC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 2350 | Phase 3 | France;United States;Canada;Spain;Turkey;South Africa;Israel;Germany;Norway;United Kingdom;New Zealand;India | ||
| 5 | EUCTR2020-002755-38-NL (EUCTR) | 23/02/2021 | 26/12/2020 | Study to evaluate efficacy and safety of inclisiran in adolescents with homozygous familial hypercholesterolemia | Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13) | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Trade Name: Leqvio 284 mg solution for injection in pre-filled syringe Product Name: inclisiran sodium Product Code: KJX839 INN or Proposed INN: Inclisiran Other descriptive name: Inclisiran sodium | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 3 | France;United States;Serbia;Slovenia;Greece;Canada;Lebanon;Viet Nam;Turkey;Netherlands;Switzerland | ||
| 6 | NCT04659863 (ClinicalTrials.gov) | February 16, 2021 | 2/12/2020 | Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia | Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13) | Familial Hypercholesterolemia - Homozygous | Drug: Inclisiran;Drug: Placebo | Novartis Pharmaceuticals | NULL | Active, not recruiting | 12 Years | 17 Years | All | 13 | Phase 3 | United States;Canada;France;Greece;Lebanon;Malaysia;Netherlands;Turkey;Slovenia;Switzerland;Vietnam |
| 7 | EUCTR2020-002755-38-GR (EUCTR) | 02/02/2021 | 13/12/2020 | Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13) | Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13) | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: inclisiran sodium Product Code: KJX839 INN or Proposed INN: inclisiran Other descriptive name: Inclisiran sodium | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 3 | France;United States;Serbia;Slovenia;Canada;Greece;Lebanon;Turkey;Netherlands;Switzerland | ||
| 8 | EUCTR2020-002755-38-SI (EUCTR) | 30/12/2020 | 02/12/2020 | Study to evaluate efficacy and safety of inclisiran in adolescents with homozygous familial hypercholesterolemia | Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13) | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: inclisiran sodium Product Code: KJX839 INN or Proposed INN: inclisiran Other descriptive name: Inclisiran sodium | Novartis Pharma AG | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 3 | Serbia;United States;Slovenia;Greece;Lebanon;Turkey;Italy;Switzerland;France;Canada;South Africa;Germany;Netherlands | ||
| 9 | NCT04681170 (ClinicalTrials.gov) | December 14, 2020 | 7/12/2020 | Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) | Phase III, Single Arm, Open Label, International, Multi Centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid Lowering Therapy | Homozygous Familial Hypercholesterolaemia (HoFH) | Drug: Lomitapide | Amryt Pharma | NULL | Recruiting | 5 Years | 17 Years | All | 45 | Phase 3 | Germany;Israel;Italy;Saudi Arabia;Spain;Tunisia |
| 10 | EUCTR2019-002278-30-IT (EUCTR) | 11/11/2020 | 21/01/2021 | A clinical trial to assess the safety and effect of the drug lomitapide in the treatment of children with the condition Homozygous Familial Hypercholesterolaemia (HoFH) who are on Stable Lipid-lowering Therapy | Phase III, single-arm, open-label, international, multi-centre study to evaluate the efficacy and safety of lomitapide in paediatric patients with Homozygous Familial Hypercholesterolaemia (HoFH) on stable lipid-lowering therapy - APH-19 | Homozygous familial hypercholesterolaemia (HoFH). A rare and life-threatening inherited disorder of lipid metabolism with an estimated prevalence of 1 per 160,000 to 300,000 in the European population. MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Trade Name: Lojuxta 5 mg hard capsules Product Name: Lojuxta 5 mg hard capsules Product Code: [PRD7255830] INN or Proposed INN: LOMITAPIDE Trade Name: Lojuxta 10 mg hard capsules Product Name: Lojuxta 10 mg hard capsules Product Code: [PRD7255977] INN or Proposed INN: LOMITAPIDE Trade Name: Lojuxta 20 mg hard capsules Product Name: Lojuxta 20 mg hard capsules Product Code: [PRD7256003] INN or Proposed INN: LOMITAPIDE Product Name: Lomitapide 2mg hard capsules Product Code: [Lomitapide 2mg hard capsules] INN or Proposed INN: LOMITAPIDE | Amryt Pharmaceuticals DAC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 45 | Phase 3 | Saudi Arabia;Spain;Turkey;Israel;Germany;Tunisia;Italy | ||
| 11 | EUCTR2019-001931-30-NL (EUCTR) | 03/11/2020 | 30/07/2020 | A trial to evaluate the Efficacy and Safety of Evinacumab in Paediatric Patients With Homozygous Familial Hypercholesterolemia | A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients with Homozygous Familial Hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 28 | Phase 1;Phase 3 | United States;Taiwan;Ukraine;Lebanon;Austria;Italy;France;Canada;Jordan;Belgium;Australia;Netherlands;Germany;Norway | ||
| 12 | NCT04455581 (ClinicalTrials.gov) | September 1, 2020 | 29/6/2020 | A Study to Determine the Safety, Tolerability, and Efficacy of SHR-1209 in Patients With Familial Hypercholesterolemia | Open-label, Single-arm, Multicentre Study to Evaluate Efficacy and Safety of SHR-1209 in Subjects With Homozygous Familial Hypercholesterolemia | Familial Hypercholesterolemia | Drug: SHR-1209 | Jiangsu HengRui Medicine Co., Ltd. | NULL | Not yet recruiting | 18 Years | 80 Years | All | 8 | Phase 2 | NULL |
| 13 | EUCTR2019-001931-30-AT (EUCTR) | 17/08/2020 | 03/03/2020 | A trial to evaluate the Efficacy and Safety of Evinacumab in Paediatric Patients With Homozygous Familial Hypercholesterolemia | A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients with Homozygous Familial Hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 1;Phase 3 | United States;Taiwan;Australia;Austria;Netherlands | ||
| 14 | NCT04515927 (ClinicalTrials.gov) | August 17, 2020 | 11/8/2020 | To Evaluate the Efficacy and Safety of JS002 in HoFH Patients | An Open-label, Single-arm Study Evaluated the Efficacy and Safety of JS002 in Patients With Homozygous Familial Hypercholesterolemia | Hyperlipemia | Drug: JS002 | Shanghai Junshi Bioscience Co., Ltd. | NULL | Active, not recruiting | 12 Years | 75 Years | All | 30 | Phase 2 | China |
| 15 | EUCTR2019-004496-39-NL (EUCTR) | 12/08/2020 | 13/11/2020 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: AAV8.TBG.hLDLR Product Code: AAV8.TBG.hLDLR | REGENXBIO Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 12 | Phase 2 | United States;Canada;Netherlands;Italy | ||
| 16 | EUCTR2019-002278-30-DE (EUCTR) | 12/08/2020 | 11/09/2019 | A clinical trial to assess the safety and effect of the drug lomitapide in the treatment of children with the condition Homozygous Familial Hypercholesterolaemia (HoFH) who are on Stable Lipid-lowering Therapy | Phase III, single-arm, open-label, international, multi-centre study to evaluate the efficacy and safety of lomitapide in paediatric patients with Homozygous Familial Hypercholesterolaemia (HoFH) on stable lipid-lowering therapy | Homozygous familial hypercholesterolaemia (HoFH). A rare and life-threatening inherited disorder of lipid metabolism with an estimated prevalence of 1 per 160,000 to 300,000 in the European population. MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Trade Name: Lojuxta 5 mg hard capsules INN or Proposed INN: LOMITAPIDE Trade Name: Lojuxta 10 mg hard capsules INN or Proposed INN: LOMITAPIDE Trade Name: Lojuxta 20 mg hard capsules INN or Proposed INN: LOMITAPIDE Product Name: Lomitapide 2mg hard capsules INN or Proposed INN: LOMITAPIDE | Amryt Pharmaceuticals DAC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 45 | Phase 3 | Saudi Arabia;Spain;Israel;Tunisia;Germany;Italy | ||
| 17 | NCT04233918 (ClinicalTrials.gov) | June 29, 2020 | 6/1/2020 | Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia | A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Drug: Evinacumab | Regeneron Pharmaceuticals | NULL | Active, not recruiting | 5 Years | 11 Years | All | 20 | Phase 3 | United States;Australia;Austria;Netherlands;Taiwan;Ukraine |
| 18 | EUCTR2019-003611-62-NO (EUCTR) | 20/03/2020 | 28/11/2019 | Randomized, Open-Label, Phase 3 Study in which patients will receive LIB003 and Evolocumab to Evaluate the Efficacy and Safety in Homozygous Familial Hypercholesterolemia Patients on Stable Lipid-Lowering Therapy. | Randomized, Open-Label, Cross-Over, Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 with Evolocumab in Homozygous Familial Hypercholesterolemia Patients on Stable Lipid-Lowering Therapy. - LIB003-003 | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Code: LIB003 INN or Proposed INN: not available Other descriptive name: LIB003 Trade Name: Repatha (evolocumab) INN or Proposed INN: EVOLOCUMAB | LIB Therapeutics, LLC | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Phase 3 | United States;Turkey;Israel;South Africa;Norway | ||
| 19 | NCT04948008 (ClinicalTrials.gov) | November 5, 2019 | 29/6/2021 | Evaluate the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia | A Seamless Phase IIb/III Clinical Study to Evaluate the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia in China | Familial Hypercholesterolemia - Homozygous;Lipid Metabolism Disorders;Proprotein Convertase Subtilisin/Kexin 9 | Biological: IBI306 | Shenzhen People's Hospital | NULL | Recruiting | 18 Years | 80 Years | All | 8 | Phase 2/Phase 3 | China |
| 20 | NCT04080050 (ClinicalTrials.gov) | September 30, 2019 | 3/9/2019 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 | Homozygous Familial Hypercholesterolemia (HoFH) | Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy | REGENXBIO Inc. | NULL | Active, not recruiting | 18 Years | N/A | All | 8 | United States;Canada;Netherlands;Italy | |
| 21 | NCT04031742 (ClinicalTrials.gov) | September 29, 2019 | 22/7/2019 | A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozygous Familial Hypercholesterolemia | Phase 2b/3 Study to Assess the Efficacy and Safety of IBI306 in Subjects With Homozygous Familial Hypercholesterolemia. | Homozygous Familial Hypercholesterolemia | Biological: IBI306 | Innovent Biologics (Suzhou) Co. Ltd. | NULL | Active, not recruiting | 18 Years | 80 Years | All | 30 | Phase 2/Phase 3 | China |
| 22 | EUCTR2015-002276-25-PT (EUCTR) | 29/07/2019 | 15/11/2018 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Norway;Netherlands;Germany;New Zealand | ||
| 23 | EUCTR2017-003170-13-AT (EUCTR) | 23/07/2019 | 11/02/2019 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 121 | Phase 3 | United States;Greece;Ukraine;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands;Japan | ||
| 24 | EUCTR2015-002276-25-SI (EUCTR) | 10/06/2019 | 06/03/2019 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 | Amgen Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Phase 3 | United States;Portugal;Taiwan;Greece;Spain;Ukraine;Russian Federation;Colombia;Italy;Switzerland;Malaysia;Australia;South Africa;Netherlands;Slovenia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Canada;Swaziland;Brazil;Belgium;Poland;Germany;Norway | ||
| 25 | NCT03933293 (ClinicalTrials.gov) | May 13, 2019 | 28/4/2019 | A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH | A Phase 2 Study to Evaluate the Safety and Efficacy of PCSK9 Inhibitor AK102 in Patients With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: AK102;Drug: Statins;Drug: Ezetimibe | Akeso | AD Pharmaceuticals Co., Ltd. | Completed | 18 Years | N/A | All | 10 | Phase 2 | China |
| 26 | NCT03814187 (ClinicalTrials.gov) | April 16, 2019 | 17/1/2019 | Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C | An Open-label Extension Trial of the Phase III Lipid-lowering Trials to Assess the Effect of Long Term Dosing of Inclisiran Given as Subcutaneous Injections in Subjects With High Cardiovascular Risk and Elevated LDL-C | ASCVD;Elevated Cholesterol;Heterozygous Familial Hypercholesterolemia;Homozygous Familial Hypercholesterolemia | Drug: Inclisiran Sodium | Novartis Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 3275 | Phase 3 | United States;Canada;Czechia;Denmark;Germany;Hungary;Netherlands;Poland;South Africa;Spain;Sweden;Ukraine;United Kingdom |
| 27 | EUCTR2017-003170-13-GB (EUCTR) | 12/03/2019 | 21/01/2019 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Greece;Ukraine;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Norway;Netherlands;Germany;Japan | ||
| 28 | EUCTR2017-003170-13-IT (EUCTR) | 18/02/2019 | 22/01/2021 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia - na | Homozygous familial hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850 MedDRA version: 20.0;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Trade Name: na Product Name: Evinacumab (REGN1500) Product Code: [REGN1500] INN or Proposed INN: Evinacumab Other descriptive name: Evinacumab Trade Name: Praluent Product Name: Praluent Product Code: [na] INN or Proposed INN: alirocumab | REGENERON PHARMACEUTICALS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 3 | United States;Czechia;Greece;Ukraine;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Norway;Germany;Netherlands;Japan | ||
| 29 | NCT03851705 (ClinicalTrials.gov) | February 6, 2019 | 7/2/2019 | A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | A Two-Part (Double-Blind Placebo Controlled/Open-Label) Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Subjects With Homozygous Familial Hypercholesterolemia (Hofh) (ORION-5) | Homozygous Familial Hypercholesterolemia | Drug: Inclisiran Sodium for injection;Drug: Placebo;Drug: Placebos | Novartis Pharmaceuticals | NULL | Completed | 18 Years | 80 Years | All | 56 | Phase 3 | Hong Kong;Israel;Russian Federation;Serbia;South Africa;Taiwan;Turkey;Ukraine;Czechia |
| 30 | EUCTR2017-003170-13-GR (EUCTR) | 01/02/2019 | 14/01/2019 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 121 | Phase 3 | United States;Czechia;Greece;Ukraine;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands;Japan | ||
| 31 | EUCTR2015-002276-25-PL (EUCTR) | 25/01/2019 | 28/03/2019 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | United States;Portugal;Taiwan;Greece;Spain;Ukraine;Russian Federation;Colombia;Italy;Malaysia;Australia;South Africa;Netherlands;Slovenia;Turkey;Austria;United Kingdom;Hungary;Czech Republic;Canada;Brazil;Poland;Belgium;Germany;Norway;New Zealand | ||
| 32 | EUCTR2017-003170-13-CZ (EUCTR) | 17/12/2018 | 11/10/2018 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 121 | Phase 3 | United States;Greece;Ukraine;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands;Japan | ||
| 33 | JPRN-JapicCTI-184099 | 30/9/2018 | 20/08/2018 | STUDY TO EVALUATE THE EFFICACY AND SAFETY OF EVINACUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF EVINACUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA | Homozygous familial hypercholesterolemia (HoFH) | Intervention name : Evinacumab INN of the intervention : Evinacumab Dosage And administration of the intervention : INTRAVENOUS Injection Control intervention name : PLACEBO INN of the control intervention : - Dosage And administration of the control intervention : INTRAVENOUS Injection | Regeneron Pharmaceuticals, Inc.(ICCC: PAREXEL International) | NULL | complete | 18 | BOTH | 57 | NA | Japan, North America, Europe, Oceania, Africa | |
| 34 | EUCTR2017-002297-39-SI (EUCTR) | 25/09/2018 | 20/06/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB | Sanofi-Aventis Recherche & Développement | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Norway;Netherlands | ||
| 35 | EUCTR2017-002297-39-NO (EUCTR) | 19/09/2018 | 15/05/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB | Sanofi-Aventis Recherche & Développement | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Netherlands;United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Norway | ||
| 36 | EUCTR2017-002297-39-BG (EUCTR) | 11/09/2018 | 31/07/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB | Sanofi-Aventis Recherche & Développement | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway | ||
| 37 | EUCTR2017-002297-39-AT (EUCTR) | 04/09/2018 | 07/06/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB | Sanofi-Aventis Recherche & Développement | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Austria;United States;Taiwan;Slovenia;Spain;Turkey;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway | ||
| 38 | EUCTR2017-002297-39-DK (EUCTR) | 03/09/2018 | 04/06/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Trade Name: Praluent Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB Trade Name: Praluent Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB | Sanofi-Aventis Recherche & Développement | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway | ||
| 39 | NCT03510715 (ClinicalTrials.gov) | August 31, 2018 | 18/4/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia | Hypercholesterolemia | Drug: Alirocumab SAR236553 (REGN727);Drug: Rosuvastatin;Drug: Ezetimibe;Drug: Cholestyramine;Drug: Nicotinic acid;Drug: Fenofibrate;Drug: Omega-3 fatty acids;Drug: Atorvastatin;Drug: Simvastatin;Drug: Fluvastatin;Drug: Pravastatin;Drug: Lovastatin | Sanofi | Regeneron Pharmaceuticals | Completed | 8 Years | 17 Years | All | 18 | Phase 3 | Brazil;Canada;Denmark;Mexico;Netherlands;Russian Federation;Slovenia;Spain;Taiwan;Turkey;Austria;France;Italy;Norway;United States |
| 40 | EUCTR2017-002297-39-ES (EUCTR) | 28/08/2018 | 09/07/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB | Sanofi-Aventis Recherche & Développement | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | Bulgaria;Argentina;Russian Federation;United States;Spain;Canada;Austria;Netherlands;Turkey;Norway;Taiwan;Brazil;Denmark;Italy;Mexico;Slovenia;France | ||
| 41 | NCT03403374 (ClinicalTrials.gov) | August 4, 2018 | 3/1/2018 | Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia | A Multicenter, Open-label, Single-arm, Study to Evaluate Safety and Tolerability of Repatha in Patients With Homozygous Familial Hypercholesterolemia (HoFH) in India | Homozygous Familial Hypercholesterolemia HoFH | Drug: evolocumab | Amgen | NULL | Completed | 12 Years | 80 Years | All | 30 | Phase 4 | India |
| 42 | EUCTR2017-002297-39-FR (EUCTR) | 13/07/2018 | 16/05/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB | Sanofi-Aventis Recherche & Développement | NULL | Not Recruiting | Female: yes Male: yes | 50 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Norway;Netherlands | ||
| 43 | EUCTR2017-002297-39-NL (EUCTR) | 02/07/2018 | 16/05/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB | Sanofi-Aventis Recherche & Développement | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway | ||
| 44 | EUCTR2017-002297-39-IT (EUCTR) | 26/06/2018 | 23/05/2018 | An Efficacy and Safety Study of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Efficacy and Safety of Alirocumab in Children and Adolescents with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: PT;Classification code 10020603;Term: Hypercholesterolaemia;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB Product Name: Alirocumab Product Code: SAR236553 INN or Proposed INN: Alirocumab Other descriptive name: ALIROCUMAB | Sanofi-Aventis Recherche & Développement | NULL | Not Recruiting | Female: yes Male: yes | 50 | Phase 3 | United States;Taiwan;Slovenia;Spain;Turkey;Austria;Russian Federation;Italy;France;Mexico;Canada;Argentina;Brazil;Denmark;Bulgaria;Netherlands;Norway | ||
| 45 | EUCTR2017-001388-19-DE (EUCTR) | 18/06/2018 | 23/01/2018 | A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol level | A randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 57 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands | ||
| 46 | EUCTR2017-001388-19-IT (EUCTR) | 11/05/2018 | 11/02/2021 | A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol level | A randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia - - | Homozygous familial hypercholesterolemia MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Trade Name: Praluent Product Name: Alirocumab Product Code: [REGN7272] INN or Proposed INN: Alirocumab Other descriptive name: Alirocumab Product Name: Evinacumab Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: Evinacumab | REGENERON PHARMACEUTICALS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 57 | Phase 3 | United States;Czechia;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands | ||
| 47 | EUCTR2017-001388-19-GR (EUCTR) | 02/05/2018 | 02/04/2018 | A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol level | A randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 65 | Phase 3 | United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Netherlands;Germany;Japan | ||
| 48 | NCT03455777 (ClinicalTrials.gov) | April 12, 2018 | 22/2/2018 | Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH) | A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of ISIS 703802 (AKCEA-ANGPTL3-LRx) Administered Subcutaneously to Patients With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: AKCEA-ANGPTL3-LRX | Akcea Therapeutics | Ionis Pharmaceuticals, Inc. | Withdrawn | 18 Years | N/A | All | 0 | Phase 2 | Canada |
| 49 | EUCTR2017-001388-19-AT (EUCTR) | 23/03/2018 | 30/01/2018 | A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol level | A randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 57 | Phase 3 | United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands | ||
| 50 | EUCTR2017-003170-13-NL (EUCTR) | 15/03/2018 | 10/01/2018 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 21.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 121 | Phase 3 | United States;Japan;Ukraine;United Kingdom;Greece;Canada;Netherlands;Austria;Czech Republic;Italy;South Africa;Australia;France;Germany | ||
| 51 | NCT03409744 (ClinicalTrials.gov) | March 13, 2018 | 8/1/2018 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Drug: evinacumab | Regeneron Pharmaceuticals | NULL | Active, not recruiting | 12 Years | N/A | All | 116 | Phase 3 | United States;Australia;Austria;Canada;Czechia;France;Greece;Italy;Japan;Netherlands;South Africa;Ukraine;Norway |
| 52 | NCT03399786 (ClinicalTrials.gov) | January 18, 2018 | 8/1/2018 | Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | A Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Drug: evinacumab;Drug: Placebo | Regeneron Pharmaceuticals | NULL | Completed | 12 Years | N/A | All | 65 | Phase 3 | United States;Australia;Austria;Canada;France;Greece;Italy;Japan;Netherlands;South Africa;Ukraine;Czechia;Germany;Turkey |
| 53 | EUCTR2017-000351-95-DE (EUCTR) | 13/12/2017 | 17/08/2017 | A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol level | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH | HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000012386;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 54 | Phase 3 | France;United States;Czech Republic;Greece;Canada;Ukraine;Turkey;Austria;South Africa;Germany;Italy | ||
| 54 | NCT03234127 (ClinicalTrials.gov) | December 6, 2017 | 26/7/2017 | Study of cardiovAscular Contrasted Phenotypes in Patients With FamIliaI hypercholesteRolemia | Study of cardiovAscular Contrasted Phenotypes in Patients With FamIliaI hypercholesteRolemia | Homozygous Familial Hypercholesterolemia | Genetic: Whole Genome Sequencing | Nantes University Hospital | NULL | Completed | 40 Years | N/A | All | 562 | N/A | France |
| 55 | EUCTR2017-000351-95-GR (EUCTR) | 08/11/2017 | 19/10/2017 | A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol level | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH | HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 54 | Phase 3 | Germany;France;United States;Czech Republic;Canada;Greece;Ukraine;Turkey;Austria;South Africa;Italy | ||
| 56 | EUCTR2017-000351-95-IT (EUCTR) | 12/10/2017 | 09/02/2021 | A study to the safety and efficacy of Alirocumab in patients with hereditary abnormal (high) cholesterol level | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to evaluate the efficacy and safety of Alirocumab in patients with Homozygous Familial Hypercholesterolemia - ODYSSEY HoFH | Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Trade Name: Praluent Product Name: R727 Product Code: [R727] INN or Proposed INN: Alirocumab Other descriptive name: Alirocumab | REGENERON PHARMACEUTICALS, INC. | NULL | Not Recruiting | Female: yes Male: yes | 55 | Phase 3 | United States;Czechia;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Denmark;South Africa;Germany | ||
| 57 | EUCTR2017-000351-95-AT (EUCTR) | 03/10/2017 | 22/08/2017 | A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol level | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH | HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 54 | Phase 3 | Greece;Canada;Austria;Czech Republic;Turkey;United States;Ukraine;Italy;South Africa;France;Germany | ||
| 58 | NCT03156621 (ClinicalTrials.gov) | October 3, 2017 | 15/5/2017 | Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Drug: Alirocumab;Drug: Placebo | Regeneron Pharmaceuticals | Sanofi | Completed | 18 Years | N/A | All | 69 | Phase 3 | United States;Austria;Canada;Czechia;France;Germany;Greece;Italy;Japan;South Africa;Taiwan;Turkey;Ukraine |
| 59 | EUCTR2016-001446-25-IT (EUCTR) | 02/10/2017 | 06/05/2021 | This is a study to investigate if gene therapy is safe in people with homozygous familial hypercholesterolemia (also called HoFH). | AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) | Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: AAV8.TBG.hLDLR Product Code: AAV8.TBG.hLDLR Product Name: prednisone Product Code: prednisone | REGENXBIO Inc. | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 1;Phase 2 | United States;Canada;Netherlands;Italy | ||
| 60 | EUCTR2016-001446-25-NL (EUCTR) | 20/09/2017 | 12/09/2016 | This is a study to investigate if gene therapy is safe in people with homozygous familial hypercholesterolemia (also called HoFH). | AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) | Adults with homozygous familial hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: AAV8.TBG.hLDLR Product Code: AAV8.TBG.hLDLR | University of Pennsylvania | NULL | Not Recruiting | Female: yes Male: yes | 12 | Phase 1;Phase 2 | United States;Canada;Netherlands;Italy | ||
| 61 | EUCTR2017-000351-95-FR (EUCTR) | 18/07/2017 | 23/06/2017 | A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol level | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH | HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 54 | Phase 3 | United States;Portugal;Serbia;Greece;Spain;Ukraine;Israel;Italy;France;Denmark;South Africa;Tunisia;Netherlands;Slovenia;Turkey;Austria;United Kingdom;Czech Republic;Canada;Belgium;Poland;Croatia;Bulgaria;Germany;Sweden | ||
| 62 | EUCTR2016-003376-49-NL (EUCTR) | 20/06/2017 | 18/01/2017 | A study to evaluate the effect of ALN-PCSSC treatment in patients with Homozygous Familial Hypercholesterolemia | An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects with Homozygous Familial Hypercholesterolemia - ORION-2 | Homozygous Familial Hypercholesterolemia MedDRA version: 19.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: ALN-PCSSC Product Code: ALN-PCSSC INN or Proposed INN: ALN-60212 Other descriptive name: ALN-60212 | The Medicines Company | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United States;South Africa;Netherlands | ||
| 63 | EUCTR2015-002276-25-NL (EUCTR) | 11/04/2017 | 22/11/2016 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 124 | Phase 3 | Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Norway;Netherlands;Germany;New Zealand | ||
| 64 | EUCTR2015-002276-25-ES (EUCTR) | 27/02/2017 | 05/08/2016 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 19.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 19.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;Italy;United Kingdom;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Netherlands;Germany;Norway;New Zealand | ||
| 65 | NCT02963311 (ClinicalTrials.gov) | December 13, 2016 | 10/11/2016 | A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: ALN-PCSSC;Drug: Standard of Care | The Medicines Company | NULL | Completed | 12 Years | N/A | All | 9 | Phase 2 | United States;Netherlands;South Africa |
| 66 | EUCTR2015-002276-25-DE (EUCTR) | 21/10/2016 | 05/04/2016 | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) | Hypercholesterolemia MedDRA version: 18.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 18.1;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;Italy;United Kingdom;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Netherlands;Germany;Norway;New Zealand | ||
| 67 | EUCTR2015-002276-25-CZ (EUCTR) | 06/10/2016 | 04/05/2016 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;United Kingdom;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Netherlands;Germany;Norway;New Zealand | ||
| 68 | EUCTR2015-004003-23-IT (EUCTR) | 28/09/2016 | 27/05/2016 | A controlled study to assess the safety, tolerability and activity of the study drug ISIS 703802 given subcutaneously to Healthy Volunteers with high triglycerides and patients with Familial Hypercholesterolemia | A Placebo-Controlled, Dose-Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of ISIS 703802, Targeting ANGPTL3, Administered Subcutaneously to Healthy Volunteers with Elevated Triglycerides andSubjects with Familial Hypercholesterolemia | Heterozygous familial hypercholesterolemia and Homozygous familial hypercholesterolemia MedDRA version: 19.0;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850 MedDRA version: 19.0;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;Classification code 10057079;Term: Heterozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: ISIS 703802 Product Code: ISIS 703802 INN or Proposed INN: ISIS 703802 Other descriptive name: ISIS 703802 | Ionis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 61 | Phase 1;Phase 2 | Canada;Netherlands;Italy | ||
| 69 | NCT02624869 (ClinicalTrials.gov) | September 10, 2016 | 22/10/2015 | Open Label Study to Evaluate Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Pediatric Subjects (10 to 17 Years of Age) With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH). | Open-label, Single-Arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) | Familial Hypercholesterolemia | Biological: evolocumab (AMG 145) | Amgen | NULL | Completed | 10 Years | 17 Years | All | 163 | Phase 3 | United States;Australia;Austria;Belgium;Brazil;Canada;Colombia;Czechia;Greece;Hungary;Italy;Malaysia;Netherlands;Norway;Poland;Portugal;Russian Federation;Slovenia;South Africa;Spain;Switzerland;Turkey;United Kingdom |
| 70 | EUCTR2015-002276-25-IT (EUCTR) | 25/07/2016 | 15/06/2021 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozyg | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg soluzione per iniezione in penna preriempita. Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: Evolocumab Trade Name: Repatha 420 mg soluzione per iniezione in cartuccia Product Name: Evolocumab Product Code: [AMG 145] INN or Proposed INN: EVOLOCUMAB | AMGEN INC. | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Norway;Netherlands;Germany;New Zealand | ||
| 71 | EUCTR2016-000411-32-NL (EUCTR) | 15/07/2016 | 17/05/2016 | A study to the safety and efficacy of REGN1500 in patients with Hereditary abnormal (high) cholesterol level | AN OPEN-LABEL, SINGLE-ARM, PROOF-OF-CONCEPT STUDY TO EVALUATE THE SAFETY AND EFFICACY OF SINGLE AND MULTIPLE DOSES OF REGN1500 IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA | Homozygous Familial Hypercholesterolemia MedDRA version: 19.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: evinacumab Product Code: REGN1500 INN or Proposed INN: EVINACUMAB Other descriptive name: EVINACUMAB Product Name: Evinacumab Product Code: REGN1500 INN or Proposed INN: EVINACUMAB Other descriptive name: EVINACUMAB Product Name: evinacumab Product Code: REGN1500 INN or Proposed INN: EVINACUMAB Other descriptive name: EVINACUMAB | Regeneron Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 8 | Phase 2 | United States;Canada;Netherlands | ||
| 72 | EUCTR2014-003806-33-GR (EUCTR) | 30/06/2016 | 24/03/2016 | A clinical study in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy conducted to assess safety, tolerability and efficacy of Lomitapide | A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients with Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy | Homozygous familial hypercholesterolemia (HoFH). MedDRA version: 18.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: lomitapide Product Code: AEGR-733 INN or Proposed INN: LOMITAPIDE Product Name: lomitapide Product Code: AEGR-733 INN or Proposed INN: LOMITAPIDE Product Name: lomitapide Product Code: AEGR-733 INN or Proposed INN: LOMITAPIDE Product Name: lomitapide Product Code: AEGR-733 INN or Proposed INN: LOMITAPIDE | Aegerion Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 45 | Phase 3 | Canada;Greece;Turkey;Austria;Israel;Germany;Italy | ||
| 73 | EUCTR2015-002276-25-AT (EUCTR) | 27/06/2016 | 12/04/2016 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Germany;Netherlands;Norway;New Zealand | ||
| 74 | EUCTR2015-002276-25-HU (EUCTR) | 06/06/2016 | 12/04/2016 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 19.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 19.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;United Kingdom;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;South Africa;Netherlands;Germany;Norway;New Zealand | ||
| 75 | JPRN-UMIN000022603 | 2016/06/06 | 04/06/2016 | Non-randomized, prospective, non-controlled study of changes of lipid parameters after introduction of evolocumab in patients with heterozygous familial hypercholesterolemia under LDL-apheresis therapy. | Non-randomized, prospective, non-controlled study of changes of lipid parameters after introduction of evolocumab in patients with heterozygous familial hypercholesterolemia under LDL-apheresis therapy. - Impact of evelocumab in homozygous familial hypercholesterolemia under LDL-apheresis. | Homozygous familial hypercholesterolemia | Subcutaneous injection of evolocumab | Kanazawa University Hospital | NULL | Complete: follow-up complete | 20years-old | 100years-old | Male and Female | 2 | Not selected | Japan |
| 76 | EUCTR2015-002276-25-GR (EUCTR) | 01/06/2016 | 16/05/2016 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;Italy;United Kingdom;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Netherlands;Germany;Norway;New Zealand | ||
| 77 | NCT02722408 (ClinicalTrials.gov) | June 2016 | 12/3/2016 | Efficacy and Safety of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1) | A Phase 2 Open-Label, Dose-Finding Study to Assess the Efficacy, Safety, and Tolerability of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid Lowering Therapy (COBALT-1) | Hypercholesteremia | Drug: Gemcabene | NeuroBo Pharmaceuticals Inc. | NULL | Completed | 17 Years | N/A | All | 8 | Phase 2 | United States;Canada;Israel |
| 78 | EUCTR2015-002276-25-BE (EUCTR) | 23/05/2016 | 13/04/2016 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Switzerland;Italy;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Norway;Netherlands;Germany;New Zealand | ||
| 79 | NCT02765841 (ClinicalTrials.gov) | May 2016 | 27/4/2016 | Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy | A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | NULL | Withdrawn | 5 Years | 17 Years | All | 0 | Phase 3 | NULL |
| 80 | NCT02651675 (ClinicalTrials.gov) | March 2016 | 4/1/2016 | A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH) | AAV8-mediated Low Density Lipoprotein Receptor (LDLR) Gene Replacement in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia (HoFH) | Biological: AAV directed hLDLR gene therapy | Regenxbio Inc. | National Heart, Lung, and Blood Institute (NHLBI) | Completed | 18 Years | N/A | All | 9 | Phase 1/Phase 2 | United States;Canada;Italy;Netherlands |
| 81 | NCT02434497 (ClinicalTrials.gov) | June 6, 2015 | 7/4/2015 | A Study to Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia | An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (Aged 6 to <18 Years) With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia (HoFH) | Drug: Rosuvastatin 20mg | AstraZeneca | NULL | Completed | 6 Years | 18 Years | All | 9 | Phase 3 | Belgium;Canada;Denmark;Israel;Malaysia;Taiwan |
| 82 | NCT02399852 (ClinicalTrials.gov) | June 2015 | 23/3/2015 | Effects of Lomitapide on Carotid and Aortic Atherosclerosis | Effects of Lomitapide on Carotid and Aortic Atherosclerosis in Patients Treated With Lomitapide in Usual Care (CAPTURE) | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | NULL | Withdrawn | 18 Years | N/A | All | 0 | N/A | NULL |
| 83 | EUCTR2014-004856-68-NO (EUCTR) | 29/04/2015 | 26/01/2015 | A 12-week, open-label , all subjects will first receive 2 weeks of placebo drug and then active medication in a dose escalating manner: each patient will first receive 50 mg, after evaluation the option exists to increase the dose to to 100 mg and after another evaluation to 200 mg active study drug, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) | A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: MBX-8025 Product Code: MBX-8025 INN or Proposed INN: MBX-8025 | CymaBay Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 8 | Phase 2 | France;Netherlands;Norway | ||
| 84 | EUCTR2014-004856-68-NL (EUCTR) | 23/04/2015 | 28/01/2015 | A 12-week, open-label , all subjects will first receive 2 weeks of placebo drug and then active medication in a dose escalating manner: each patient will first receive 50 mg, after evaluation the option exists to increase the dose to to 100 mg and after another evaluation to 200 mg active study drug, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) | A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: MBX-8025 Product Code: MBX-8025 INN or Proposed INN: MBX-8025 | CymaBay Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 8 | Phase 2 | France;Norway;Netherlands | ||
| 85 | NCT02472535 (ClinicalTrials.gov) | April 2015 | 22/5/2015 | Study to Evaluate the Effects of MBX-8025 in Patients With HoFH | A 12-week, Open-label, Dose-escalating, Phase 2 Study to Evaluate the Effects of MBX-8025 in Patients With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Other: Run-In Period: Placebo;Drug: MBX-8025 50 mg (Dose Escalation Period 1);Drug: MBX-8025 50 mg or 100 mg (Dose Escalation Period 2);Drug: MBX-8025 50 mg, 100 mg or 200 mg (Dose Escalation Period 3) | CymaBay Therapeutics, Inc. | NULL | Completed | 18 Years | N/A | Both | 13 | Phase 2 | Canada;France;Netherlands;Norway |
| 86 | EUCTR2014-004746-99-DK (EUCTR) | 27/03/2015 | 27/03/2015 | A study to evluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia. | An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH). MedDRA version: 19.0;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Film-Coated Tablet Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: Rosuvastatin calcium | AstraZeneca AB | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 3 | United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Netherlands;Germany;Sweden | ||
| 87 | EUCTR2014-004856-68-FR (EUCTR) | 13/03/2015 | 19/05/2015 | A 12-week, open-label , all subjects will first receive 2 weeks of placebo drug and then active medication in a dose escalating manner: each patient will first receive 50 mg, after evaluation the option exists to increase the dose to to 100 mg and after another evaluation to 200 mg active study drug, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) | A 12-week, open-label, dose-escalating, phase 2 study to evaluate the effects of MBX-8025 in patients with Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: MBX-8025 Product Code: MBX-8025 INN or Proposed INN: MBX-8025 | CymaBay Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 8 | Phase 2 | France;Norway;Netherlands | ||
| 88 | EUCTR2014-004746-99-BE (EUCTR) | 23/02/2015 | 18/12/2014 | A study to evluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia. | An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH).;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Film-Coated Tablet Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: Rosuvastatin calcium | AstraZeneca AB | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 3 | United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Netherlands;Germany;Sweden | ||
| 89 | NCT02265952 (ClinicalTrials.gov) | February 4, 2015 | 10/10/2014 | Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | An Open-Label, Single-Arm, Proof-of-Concept Study to Evaluate the Safety and Efficacy of Single and Multiple Doses of REGN1500 in Patients With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Drug: REGN1500 250 mg SC/15 mg/kg IV/450 mg SC | Regeneron Pharmaceuticals | NULL | Completed | 18 Years | N/A | All | 9 | Phase 2 | United States;Canada;Netherlands |
| 90 | EUCTR2014-000972-24-DK (EUCTR) | 17/12/2014 | 25/09/2014 | A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia | A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.0;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin | AstraZeneca AB | NULL | Not Recruiting | Female: yes Male: yes | 25 | United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Germany;Netherlands;Sweden | |||
| 91 | EUCTR2014-000972-24-DE (EUCTR) | 17/11/2014 | 26/09/2014 | A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia | A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin | AstraZeneca AB | NULL | Not Recruiting | Female: yes Male: yes | 25 | United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Denmark;Australia;South Africa;Germany;Netherlands;Norway;Sweden | |||
| 92 | NCT02226198 (ClinicalTrials.gov) | November 2014 | 14/8/2014 | A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia | A Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (Aged 6 to <18 Years) With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia (HoFH) | Drug: Rosuvastatin 20mg;Drug: Placebo | AstraZeneca | NULL | Completed | 6 Years | 17 Years | All | 20 | Phase 3 | Belgium;Canada;Denmark;Israel;Malaysia;Netherlands;Taiwan;Germany;Lebanon;Sweden;United States |
| 93 | EUCTR2014-000972-24-BE (EUCTR) | 27/10/2014 | 11/09/2014 | A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia | A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin | AstraZeneca AB | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 3 | United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden | ||
| 94 | EUCTR2014-000972-24-NL (EUCTR) | 20/10/2014 | 05/08/2014 | A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia | A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin | AstraZeneca AB | NULL | Not Recruiting | Female: yes Male: yes | 25 | United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden | |||
| 95 | EUCTR2014-000972-24-SE (EUCTR) | 20/08/2014 | 30/06/2014 | A study to evaluate the effect of Rosuvastatin on fatty acids in Children and Adolescents with Homozygous Familial Hypercholesterolemia | A Randomized, Double blind, Placebo controlled, Multi center, Cross over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) - HYDRA | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 17.1;Level: LLT;Classification code 10054380;Term: Familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Tablets (calcium rosuvastatin) Film-Coated tablets Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: calcium rosuvastatin | AstraZeneca AB | NULL | Not Recruiting | Female: yes Male: yes | 25 | United States;Taiwan;Hong Kong;Israel;United Kingdom;Czech Republic;Belgium;Australia;Denmark;South Africa;Germany;Netherlands;Norway;Sweden | |||
| 96 | NCT02173158 (ClinicalTrials.gov) | April 2, 2014 | 23/6/2014 | Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy | A Phase 3, Single-Arm, Open-Label, Multicenter Study to Evaluate the Efficacy and Safety of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Concurrent Lipid-Lowering Therapy | Familial Hypercholesterolemia - Homozygous | Drug: lomitapide | Aegerion Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 9 | Phase 3 | Japan |
| 97 | NCT02135705 (ClinicalTrials.gov) | March 18, 2014 | 10/4/2014 | LOWER: Lomitapide Observational Worldwide Evaluation Registry | LOWER: Lomitapide Observational Worldwide Evaluation Registry | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Amryt Pharma | NULL | Recruiting | N/A | N/A | All | 300 | United States;Argentina;Canada;France;Greece;Italy;Netherlands;United Kingdom | |
| 98 | EUCTR2011-005400-15-IT (EUCTR) | 19/12/2013 | 22/04/2013 | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | Homozygous Familial Hypercholesterolemia MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 INN or Proposed INN: NA Other descriptive name: AMG 145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 125 | Phase 2 | France;Czech Republic;Canada;Poland;Belgium;Brazil;Malaysia;Australia;South Africa;Netherlands;Norway;New Zealand;Japan;United States;Hong Kong;Greece;Spain;Turkey;Austria;Israel;United Kingdom;Italy | ||
| 99 | EUCTR2011-005399-40-NL (EUCTR) | 24/09/2013 | 03/07/2013 | A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemia | A two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia | Homozygous familial hypercholesterolaemia MedDRA version: 16.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Other descriptive name: AMG 145 | Amgen Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 59 | Phase 2/3 | United States;Hong Kong;Spain;Lebanon;Turkey;Italy;France;Czech Republic;Canada;Brazil;Belgium;Malaysia;South Africa;Netherlands;New Zealand | ||
| 100 | EUCTR2011-005400-15-NL (EUCTR) | 24/09/2013 | 04/07/2013 | A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutations | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | Familial Hypercholesterolemia MedDRA version: 18.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Other descriptive name: AMG 145 Product Name: AMG 145 Other descriptive name: AMG145 Product Name: AMG 145 Other descriptive name: AMG145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 2;Phase 3 | United States;Hong Kong;Greece;Spain;Lebanon;Turkey;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;Australia;South Africa;Netherlands;New Zealand;Japan | ||
| 101 | EUCTR2012-002434-37-IT (EUCTR) | 16/07/2013 | 05/06/2013 | n.a. | A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-WeekStudy to Assess the Efficacy and Tolerability of Anacetrapib When Added to OngoingLipid-Lowering Therapy in Adult Patients with Homozygous FamilialHypercholesterolemia (HoFH) - n.a. | Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 14.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: Anacetrapib Product Code: MK-0859 INN or Proposed INN: ANACETRAPIB Other descriptive name: ANACETRAPIB | Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc | NULL | Not Recruiting | Female: yes Male: yes | 45 | Phase 3 | United States;Czech Republic;Canada;Malaysia;Brazil;Israel;South Africa;Norway;United Kingdom;Italy | ||
| 102 | EUCTR2011-005399-40-ES (EUCTR) | 24/06/2013 | 09/04/2013 | A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemia | A two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia | Homozygous familial hypercholesterolaemia MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 INN or Proposed INN: n/a Other descriptive name: AMG 145 | Amgen Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 67 | Phase 2/3 | United States;Hong Kong;Spain;Turkey;Austria;France;Czech Republic;Canada;Belgium;Brazil;Malaysia;South Africa;New Zealand | ||
| 103 | EUCTR2011-005400-15-ES (EUCTR) | 18/06/2013 | 09/04/2013 | A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with Severe Familial Hypercholesterolemia | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | Homozygous Familial Hypercholesterolemia MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 INN or Proposed INN: n/a Other descriptive name: AMG 145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 125 | Phase 2 | United States;Hong Kong;Greece;Spain;Turkey;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Malaysia;Poland;Belgium;Brazil;Australia;South Africa;Netherlands;Norway;Japan;New Zealand | ||
| 104 | EUCTR2012-002434-37-CZ (EUCTR) | 13/06/2013 | 28/12/2012 | n/a | A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-WeekStudy to Assess the Efficacy and Tolerability of Anacetrapib When Added to OngoingLipid-Lowering Therapy in Adult Patients with Homozygous FamilialHypercholesterolemia (HoFH) with a 52-Week Open-Label Extension | Homozygous Familial Hypercholesterolemia (HoFH);Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: MK-0859 Product Code: (4S,5R)-5-[3,5-bis(trifluoromethyl)phenyl]-3-{[4’- | Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc | NULL | Not Recruiting | Female: yes Male: yes | 45 | United States;Czech Republic;Canada;Malaysia;Australia;Israel;South Africa;Norway;United Kingdom | |||
| 105 | EUCTR2011-005399-40-IT (EUCTR) | 08/06/2013 | 23/04/2013 | A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemia | A two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia | Homozygous familial hypercholesterolaemia MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 INN or Proposed INN: NA Other descriptive name: AMG 145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 67 | Phase 2/3 | United States;Hong Kong;Spain;Turkey;Austria;Italy;France;Czech Republic;Canada;Belgium;Brazil;Malaysia;South Africa;Netherlands;New Zealand | ||
| 106 | NCT01841684 (ClinicalTrials.gov) | June 2013 | 24/4/2013 | Efficacy and Tolerability of Anacetrapib Added to Ongoing Lipid-Lowering Therapy in Adult Participants With Homozygous Familial Hypercholesterolemia (HoFH) (MK-0859-042) | A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-Week Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Lipid-Lowering Therapy in Adult Patients With Homozygous Familial Hypercholesterolemia (HoFH) | Hyperlipoproteinemia Type II;Homozygous Familial Hypercholesterolemia | Drug: Anacetrapib;Drug: Placebo | Merck Sharp & Dohme Corp. | NULL | Terminated | 18 Years | N/A | Both | 2 | Phase 3 | United States;Canada;Germany;Israel;Italy;United Kingdom;Norway |
| 107 | EUCTR2011-005400-15-CZ (EUCTR) | 30/05/2013 | 27/02/2013 | A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutations | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | Familial Hypercholesterolemia MedDRA version: 19.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: evolocumab Product Code: AMG 145 INN or Proposed INN: Evolocumab Other descriptive name: AMG 145 Trade Name: Repatha 140 mg solution for injection in pre-filled pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: Evolocumab Other descriptive name: AMG145 Product Name: Evolocumab Product Code: AMG 145 Other descriptive name: AMG145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 2;Phase 3 | United States;Hong Kong;Greece;Spain;Lebanon;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;South Africa;Netherlands;New Zealand;Japan | ||
| 108 | EUCTR2012-002434-37-GB (EUCTR) | 23/05/2013 | 23/11/2012 | n/a | A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-Week Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Lipid-Lowering Therapy in Adult Patients with Homozygous Familial Hypercholesterolemia (HoFH) with a 52-Week Open-Label Extension - Anacetrapib in Patients with Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia (HoFH);Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: MK-0859 Product Code: (4S,5R)-5-[3,5-bis(trifluoromethyl)phenyl]-3-{[4’- | Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc | NULL | Not Recruiting | Female: yes Male: yes | 45 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Czech Republic;Canada;Malaysia;Australia;Israel;South Africa;Norway;Italy;United Kingdom | ||
| 109 | EUCTR2011-005400-15-GB (EUCTR) | 22/05/2013 | 04/03/2013 | A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutations | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | Familial Hypercholesterolemia MedDRA version: 19.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Other descriptive name: AMG 145 Product Name: AMG 145 Other descriptive name: AMG145 Product Name: AMG 145 Other descriptive name: AMG145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 300 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Hong Kong;Greece;Spain;Lebanon;Turkey;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;Australia;South Africa;Netherlands;New Zealand;Japan | ||
| 110 | EUCTR2011-005400-15-AT (EUCTR) | 07/05/2013 | 20/03/2013 | A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutations | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | Homozygous Familial Hypercholesterolemia MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Other descriptive name: AMG 145 | Amgen Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 125 | United States;Hong Kong;Greece;Spain;Turkey;Austria;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Brazil;Malaysia;Australia;South Africa;Norway;New Zealand;Japan | |||
| 111 | EUCTR2011-005399-40-CZ (EUCTR) | 03/05/2013 | 26/02/2013 | A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemia | A two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia | Homozygous familial hypercholesterolaemia MedDRA version: 16.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Other descriptive name: AMG 145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 59 | Phase 2;Phase 3 | United States;Hong Kong;Spain;Lebanon;Turkey;Italy;France;Czech Republic;Canada;Brazil;Belgium;Malaysia;South Africa;Netherlands;New Zealand | ||
| 112 | EUCTR2012-002434-37-NO (EUCTR) | 21/11/2012 | 26/11/2012 | n/a | A Worldwide, Multicenter, Double-Blind, Randomized, Placebo-Controlled, 12-WeekStudy to Assess the Efficacy and Tolerability of Anacetrapib When Added to OngoingLipid-Lowering Therapy in Adult Patients with Homozygous FamilialHypercholesterolemia (HoFH) with a 52-Week Open-Label Extension | Homozygous Familial Hypercholesterolemia (HoFH);Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: MK-0859 Product Code: (4S,5R)-5-[3,5-bis(trifluoromethyl)phenyl]-3-{[4’- | Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc | NULL | Not Recruiting | Female: yes Male: yes | 45 | United States;Czech Republic;Canada;Malaysia;Australia;Israel;South Africa;Norway;United Kingdom | |||
| 113 | EUCTR2011-005400-15-GR (EUCTR) | 05/09/2012 | 09/08/2012 | A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutations | A Multicenter, Single Arm, Open-label Study to Assess the Long-term Safety and Efficacy of AMG 145 on LDL-C in Subjects With Homozygous Familial Hypercholesterolemia or PCSK9 Mutations | Homozygous Familial Hypercholesterolemia MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Other descriptive name: AMG 145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 75 | Phase 2 | United States;Czech Republic;Canada;Greece;Spain;Belgium;South Africa;Netherlands;Italy;United Kingdom | ||
| 114 | NCT01588496 (ClinicalTrials.gov) | April 5, 2012 | 27/2/2012 | Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities | 2-part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia. Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia. Part B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Biological: Evolocumab;Drug: Placebo | Amgen | NULL | Completed | 12 Years | 80 Years | All | 58 | Phase 2/Phase 3 | United States;Belgium;Canada;Czechia;France;Hong Kong;Italy;Lebanon;Netherlands;New Zealand;South Africa;Spain;Czech Republic |
| 115 | EUCTR2011-005400-15-BE (EUCTR) | 22/03/2012 | 07/02/2012 | A clinical study to assess the safety and efficacy of AMG 145 on low density cholesterol in subjects with homozygous familial hypercholesterolemia or PCSK9 mutations | A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial Hypercholesterolemia - TAUSSIG - Trial Assessing long term USe of PCSK9 Inhibition in Subjects wIth Genetic LDL | Familial Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000012386;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Other descriptive name: AMG 145 Product Name: AMG 145 Other descriptive name: AMG145 Product Name: AMG 145 Other descriptive name: AMG145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 300 | Phase 2;Phase 3 | United States;Hong Kong;Greece;Spain;Lebanon;Turkey;Austria;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;Australia;South Africa;Netherlands;New Zealand;Japan | ||
| 116 | EUCTR2011-005399-40-BE (EUCTR) | 09/03/2012 | 07/02/2012 | A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemia | A two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia | Homozygous familial hypercholesterolaemia MedDRA version: 14.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 Other descriptive name: AMG 145 | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 59 | Phase 2;Phase 3 | United States;Hong Kong;Spain;Lebanon;Turkey;Italy;France;Czech Republic;Canada;Malaysia;Belgium;Brazil;South Africa;Netherlands;New Zealand | ||
| 117 | EUCTR2011-003998-28-NL (EUCTR) | 28/02/2012 | 14/11/2011 | Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia | Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE | homozygous familial hypercholesterolemia MedDRA version: 16.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant Human Apolipoprotein A-I/Phospholipids Complex Product Code: CER-001 Other descriptive name: recombinant human apolipoprotein A-I Other descriptive name: sphingomyelin Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol) | Cerenis Therapeutics | NULL | Not Recruiting | Female: yes Male: yes | 30 | France;United States;Canada;Netherlands;Italy;United Kingdom | |||
| 118 | EUCTR2011-003998-28-IT (EUCTR) | 16/02/2012 | 08/03/2012 | Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia | Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE | Homozygous familial hypercholesterolemia MedDRA version: 15.1;Level: SOC;Classification code 10027433;Term: Metabolism and nutrition disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant Human Apolipoprotein Product Code: CER-001 Other descriptive name: Recombinant Human Apolipoprotein A-I Other descriptive name: Sfingomielina Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol) | CERENIS THERAPEUTICS SA | NULL | Not Recruiting | Female: yes Male: yes | 30 | United States;Canada;Netherlands;United Kingdom;Italy | |||
| 119 | EUCTR2011-003998-28-GB (EUCTR) | 24/01/2012 | 19/10/2011 | Effect of CER-001 on blockage of arteries (plaque) in subjects with homozygous familial hypercholesterolemia | Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) - MODE | homozygous familial hypercholesterolemia MedDRA version: 15.0;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 10027433 - Metabolism and nutrition disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Recombinant Human Apolipoprotein A-I/Phospholipids Complex Product Code: CER-001 INN or Proposed INN: recombinant human apolipoprotein a1 Other descriptive name: recombinant human apolipoprotein A-I INN or Proposed INN: sphingomyelin Other descriptive name: sphingomyelin INN or Proposed INN: DPPG Other descriptive name: 1,2-Dihexadecanoyl-sn-Glycero-3-Phospho-(1-raac-glycerol) | Cerenis Therapeutics | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Netherlands;Italy;United Kingdom | ||
| 120 | NCT01412034 (ClinicalTrials.gov) | November 2011 | 5/8/2011 | Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects | Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-label Study of the Effects of CER-001 on Plaque Volume in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: CER-001 | Cerenis Therapeutics, SA | NULL | Completed | 12 Years | N/A | Both | 23 | Phase 2 | United States;Canada;Italy;Netherlands;United Kingdom |
| 121 | EUCTR2010-023742-79-IT (EUCTR) | 25/03/2011 | 21/02/2011 | A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND | A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor ‘lomitapide’ (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia - ND | Homozygous Familial Hypercholesterolemia MedDRA version: 9.1;Level: LLT;Classification code 10057080 | Product Name: Lomitapide Product Code: AEGR-733 Product Name: Lomitapide Product Code: AEGR-733 | AEGERION PHARMACEUTICALS | NULL | Not Recruiting | Female: yes Male: yes | 23 | Phase 3 | Italy | ||
| 122 | NCT00943306 (ClinicalTrials.gov) | October 29, 2009 | 21/7/2009 | Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia | A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor 'Lomitapide' (LOMITAPIDE) in Patients With Homozygous Familial Hypercholesterolemia | Familial Hypercholesterolemia | Drug: lomitapide | Aegerion Pharmaceuticals, Inc. | NULL | Completed | 18 Years | N/A | All | 19 | Phase 3 | United States;Canada;Italy;South Africa |
| 123 | EUCTR2008-007058-36-IT (EUCTR) | 25/06/2009 | 03/06/2009 | A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - ND | A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients with Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy - ND | Homozygous Familial Hypercholesterolemia MedDRA version: 9.1;Level: LLT;Classification code 10048373;Term: Hypercholesterolaemia aggravated | Product Code: AEGR-733 Product Code: AEGR-733 | Aegerion Pharmaceuticals | NULL | Not Recruiting | Female: yes Male: yes | 25 | Phase 3 | Italy | ||
| 124 | NCT00730236 (ClinicalTrials.gov) | December 2007 | 6/8/2008 | A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH) | A Phase III Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor AEGR-733 in Patients With Homozygous Familial Hypercholesterolemia on Current Lipid-lowering Therapy | Homozygous Familial Hypercholesterolemia | Drug: AEGR-733 | Aegerion Pharmaceuticals, Inc. | FDA Office of Orphan Products Development | Completed | 18 Years | N/A | All | 29 | Phase 3 | United States;Canada;Italy;South Africa |
| 125 | EUCTR2005-003449-15-GB (EUCTR) | 12/07/2007 | 09/03/2007 | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects - RADICHOL I | Reduction of low density lipoprotein C (LDL-C) in Homozygous Familial Hypercholesterolemia (HoFH) MedDRA version: 9.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia | Product Name: ISIS 301012 Product Code: ISIS 301012 Other descriptive name: ISIS 301012 phosphorothioate oligonucleotide | Genzyme Europe B.V. | NULL | Not Recruiting | Female: yes Male: yes | 50 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United Kingdom | ||
| 126 | NCT00607373 (ClinicalTrials.gov) | July 2007 | 22/1/2008 | Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia | A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Mipomersen as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects | Lipid Metabolism, Inborn Errors;Hypercholesterolemia, Autosomal Dominant;Hyperlipidemias;Metabolic Diseases;Hyperlipoproteinemia Type II;Metabolism, Inborn Errors;Genetic Diseases, Inborn;Infant, Newborn, Diseases;Metabolic Disorder;Congenital Abnormalities;Hypercholesterolemia;Hyperlipoproteinemias;Dyslipidemias;Lipid Metabolism Disorders | Drug: mipomersen;Drug: Placebo | Kastle Therapeutics, LLC | Ionis Pharmaceuticals, Inc. | Completed | 12 Years | N/A | All | 51 | Phase 3 | United States;Brazil;Canada;Singapore;South Africa;Taiwan;United Kingdom;Netherlands |
| 127 | EUCTR2005-003626-26-GB (EUCTR) | 14/06/2006 | 01/11/2005 | A double-blind, placebo-controlled, randomized study to evaluate the efficacy and safety of TAK-475 or placebo when co-administered with current lipid-lowering therapy in subjects with homozygous familial hypercholesterolemia | A double-blind, placebo-controlled, randomized study to evaluate the efficacy and safety of TAK-475 or placebo when co-administered with current lipid-lowering therapy in subjects with homozygous familial hypercholesterolemia | Treatment of primary dyslipidemia MedDRA version: 8;Level: PT;Classification code 10058108 | Product Name: TAK-475 Product Code: TAK-475 | Takeda Europe R&D Centre Ltd | NULL | Not Recruiting | Female: yes Male: yes | 40 | United Kingdom | |||
| 128 | NCT00704535 (ClinicalTrials.gov) | March 2006 | 23/6/2008 | Evaluation of the Safety, Tolerability and Efficacy of Ezetimibe on a Select Population of Filipinos With Hypercholesterolemia (Study P04748)(COMPLETED) | Post-marketing Surveillance of the Safety, Tolerability and Efficacy of Ezetimibe Among Filipino Patients | Primary Hypercholesterolemia;Homozygous Familial Hypercholesterolemia | Drug: Ezetimibe | Organon and Co | NULL | Completed | 18 Years | N/A | All | 4105 | Philippines | |
| 129 | NCT00280995 (ClinicalTrials.gov) | January 2006 | 20/1/2006 | Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy | A Phase 2, Open-Label, Dose Escalation Study to Assess the Safety and Efficacy of ISIS 301012 as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects | Hypercholesterolemia, Familial | Drug: ISIS 301012 | Kastle Therapeutics, LLC | Ionis Pharmaceuticals, Inc. | Completed | 18 Years | N/A | Both | 12 | Phase 2 | United States;Netherlands |
| 130 | NCT00263081 (ClinicalTrials.gov) | November 2005 | 6/12/2005 | Efficacy of Lapaquistat Acetate Co-Administered With Current Lipid-Lowering Treatment on Blood Cholesterol Levels in Subjects With Homozygous Familial Hypercholesterolemia | A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Efficacy and Safety of TAK-475 or Placebo When Co-administered With Current Lipid-lowering Therapy in Subjects With Homozygous Familial Hypercholesterolemia. | Hypercholesterolemia | Drug: Lapaquistat acetate and current lipid-lowering treatment;Drug: Current lipid-lowering treatment | Takeda | NULL | Terminated | 8 Years | N/A | Both | 44 | Phase 3 | United States;Canada;France;Israel;Poland;United Kingdom |
| 131 | NCT00079846 (ClinicalTrials.gov) | September 2003 | 17/3/2004 | Implitapide in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy | Safety and Efficacy Study of Implitapide Compared With Placebo in Patients With Homozygous Familial Hypercholesterolemia (HoFH) on Maximal Concurrent Lipid-Lowering Therapy | Familial Hypercholesterolemia | Drug: Implitapide | Medical Research Laboratories International | NULL | Terminated | 8 Years | 70 Years | Both | 60 | Phase 2 | United States;Canada;Israel;Netherlands;Norway |
| 132 | NCT01556906 (ClinicalTrials.gov) | June 2003 | 7/3/2012 | Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor | A Phase II Open Label, Dose-Escalation Study to Determine the Safety, Tolerability and Efficacy of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor BMS-201038 in Patients With Homozygous Familial Hypercholeterolemia | Homozygous Familial Hypercholesterolemia | Drug: Lomitapide | Aegerion Pharmaceuticals, Inc. | University of Pennsylvania;Doris Duke Charitable Foundation | Completed | 13 Years | N/A | All | 6 | Phase 2 | United States |
| 133 | NCT00092833 (ClinicalTrials.gov) | July 2002 | 23/9/2004 | Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED) | An Open-Label, Worldwide, Treatment Use Study to Provide Ezetimibe 10 Mg/Day to Patients With Homozygous Familial Hypercholesterolemia or Homozygous Sitosterolemia | Hypercholesterolemia, Familial;Lipid Metabolism, Inborn Errors | Drug: Comparator: ezetimibe | Organon and Co | NULL | Terminated | 8 Years | N/A | All | 49 | Phase 3 | United States |
| 134 | NCT01878604 (ClinicalTrials.gov) | October 2001 | 7/6/2013 | Gene Analysis and Treatment Optimization in Chinese Homozygous Familial Hypercholesterolemia | The Study of Gene Analysis and Treatment Optimization in Chinese Homozygous Familial Hypercholesterolemia | Homozygous Familial Hypercholesterolemia | Genetic: Gene analysis;Other: Historical data of lipid-lowering drug administration;Other: Historical data of plasma lipids, xanthoma changes | Central South University | NULL | Completed | N/A | N/A | All | 5 | N/A | China |
| 135 | NCT03885921 (ClinicalTrials.gov) | October 25, 2000 | 20/3/2019 | Safety and Tolerability Study of Ezetimibe (SCH 058235/MK-0653) Plus Atorvastatin or Simvastatin in Homozygous Familial Hypercholesterolemia (P01417/MK-0653-019) | Long-Term, Open-Label, Safety and Tolerability Study of SCH 58235 in Addition to Atorvastatin or Simvastatin in the Therapy of Homozygous Familial Hypercholesterolemia | Hypercholesterolemia | Drug: Ezetimibe;Drug: Atorvastatin;Drug: Simvastatin | Organon and Co | NULL | Completed | 12 Years | N/A | All | 44 | Phase 3 | NULL |
| 136 | NCT03884452 (ClinicalTrials.gov) | May 3, 2000 | 6/3/2019 | Ezetimibe (SCH 58235) Taken With Either Atorvastatin or Simvastatin in Participants With Familial Hypercholesterolemia (MK-0653-018) | A Phase III Efficacy And Safety Study of Ezetimibe (SCH58235) 10 mg in Addition to Atorvastatin or Simvastatin in the Therapy of Homozygous Familial Hypercholesterolemia | Familial Hypercholesterolemia | Drug: Atorvastatin;Drug: Simvastatin;Drug: Ezetimibe;Drug: Placebo for Ezetimibe | Organon and Co | NULL | Completed | 12 Years | N/A | All | 50 | Phase 3 | NULL |
| 137 | EUCTR2017-001388-19-FR (EUCTR) | 24/01/2018 | A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol level | A randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals Inc. | NULL | NA | Female: yes Male: yes | 57 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands | |||
| 138 | EUCTR2011-005399-40-Outside-EU/EEA (EUCTR) | 17/12/2014 | A clinical study to assess the safety, tolerability and efficacy of AMG 145 in subjects with homozygous familial hypercholesterolemia | A two part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial HypercholesterolemiaPart B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia - TESLA | Homozygous familial hypercholesterolaemia MedDRA version: 17.1;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: AMG 145 Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | NA | Female: yes Male: yes | 59 | Phase 2/3 | South Africa;Turkey;United States;Brazil;Canada;Hong Kong;Lebanon;Malaysia;New Zealand | |||
| 139 | EUCTR2015-002276-25-GB (EUCTR) | 05/04/2016 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | NA | Female: yes Male: yes | 115 | Phase 3 | Portugal;Czechia;Slovenia;Greece;Spain;Turkey;Austria;Russian Federation;Colombia;United Kingdom;Italy;Czech Republic;Hungary;Canada;Poland;Belgium;Brazil;Australia;South Africa;Norway;Germany;Netherlands;New Zealand | |||
| 140 | EUCTR2017-000351-95-CZ (EUCTR) | 28/07/2017 | A study to the safety and efficacy of alirocumab in patients with Hereditary abnormal (high) cholesterol level | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALIROCUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA - ODYSSEY HoFH | HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA MedDRA version: 20.0;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000012386;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 54 | Phase 3 | Serbia;Portugal;United States;Slovenia;Greece;Spain;Ukraine;Turkey;Austria;Israel;Italy;United Kingdom;Czech Republic;Canada;Poland;Belgium;Croatia;Denmark;South Africa;Bulgaria;Netherlands;Germany;Tunisia;Sweden | |||
| 141 | EUCTR2015-002276-25-NO (EUCTR) | 11/04/2016 | Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia | Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE | Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 10057100;Term: Homozygous familial hypercholesterolaemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057099;Term: Heterozygous familial hypercholesterolaemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB Trade Name: Repatha 420 mg solution for injection in cartridge Product Name: Evolocumab Product Code: AMG 145 INN or Proposed INN: EVOLOCUMAB | Amgen Inc | NULL | Not Recruiting | Female: yes Male: yes | 115 | Phase 3 | South Africa;Netherlands;Norway;Portugal;Slovenia;Greece;Spain;Turkey;Austria;Colombia;Italy;United Kingdom;Czech Republic;Hungary;Canada;Poland;Brazil;Belgium;Australia;Germany;New Zealand | |||
| 142 | EUCTR2020-004394-49-DE (EUCTR) | 27/05/2021 | Study to Evaluate the Long-Term Efficacy and Safety of the Investigational Drug LIB003 for the Reduction of Cholesterol in Patients with Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease. | Open-Label Extension Phase 3 Study to Evaluate the Long-Term Efficacy and Safety of LIB003 in Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease, on Stable Lipid-Lowering Therapy Requiring Additional Low-Density Lipoprotein Cholesterol Reduction (LIBerate-OLE) - LIBerate-OLE | Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or at High Risk for Cardiovascular Disease MedDRA version: 20.0;Level: LLT;Classification code 10057079;Term: Heterozygous familial hypercholesterolemia;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850 MedDRA version: 20.1;Classification code 10007648;Term: Cardiovascular disease, unspecified;System Organ Class: 100000004849;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: LIB003 Product Code: lerodalcibep INN or Proposed INN: Lerodalcibep Other descriptive name: LIB003 | LIB Therapeutics, LLC | NULL | NA | Female: yes Male: yes | 2000 | Phase 3 | United States;France;Canada;Spain;Turkey;South Africa;Israel;Norway;Germany;United Kingdom;New Zealand;India | |||
| 143 | EUCTR2017-001388-19-CZ (EUCTR) | 01/02/2018 | A study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high) cholesterol level | A randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 57 | Phase 3 | United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands | |||
| 144 | EUCTR2017-003170-13-FR (EUCTR) | 26/10/2018 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia | Homozygous familial hypercholesterolemia MedDRA version: 20.1;Level: LLT;Classification code 10020604;Term: Hypercholesterolemia;System Organ Class: 100000004861 ;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: Evinacumab (REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descriptive name: EVINACUMAB Trade Name: Praluent INN or Proposed INN: ALIROCUMAB Other descriptive name: ALIROCUMAB | Regeneron Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 100 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Greece;Ukraine;Turkey;Austria;United Kingdom;Italy;France;Czech Republic;Canada;Australia;South Africa;Norway;Netherlands;Germany;Japan | |||
| 145 | EUCTR2014-004746-99-SE (EUCTR) | 23/12/2014 | A study to evluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia. | An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH) | Hypercholesterolemia - The current trial will study children with Homozygous Familial Hypercholesterolemia (HoFH).;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Crestor Film-Coated Tablet Product Name: Crestor Product Code: ZD4522 INN or Proposed INN: Rosuvastatin calcium | AstraZeneca AB | NULL | Not Recruiting | Female: yes Male: yes | 20 | Phase 3 | United States;Taiwan;Canada;Malaysia;Belgium;Lebanon;Denmark;Israel;Netherlands;Germany;Sweden |