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 : 142 / 薬物数 : 114 - (DrugBank : 29) / 標的遺伝子数 : 9 - 標的パスウェイ数 : 18
Showing 1 to 10 of 142 diseases
| 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 | 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. Study to Evaluate the Long-Term Efficacy and Safety of the Investigational Drug LIB003 for the Reduc ... | 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 Open-Label Extension Phase 3 Study to Evaluate the Long-Term Efficacy and Safety of LIB003 in Patien ... | 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] Patients With Homozygous and Heterozygous Familial Hypercholesterolemia, Cardiovascular Disease, or ... | Product Name: LIB003 Product Code: lerodalcibep INN or Proposed INN: Lerodalcibep Other descriptive name: LIB003 Product Name: LIB003 Product Code: lerodalcibep INN or Proposed INN: Lerodalcibep Other descriptive nam ... | 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 France;United States;Canada;Spain;Turkey;South Africa;Israel;Germany;Norway;United Kingdom;New Zeala ... | ||
| 2 | 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 Study to evaluate efficacy and safety of inclisiran in adolescents with homozygous familial hypercho ... | 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 ... | 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] Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 1005708 ... | Product Name: inclisiran sodium Product Code: KJX839 INN or Proposed INN: inclisiran Other descriptive name: Inclisiran sodium Product Name: inclisiransodium Product Code: KJX839 INN or Proposed INN: inclisiran Other descriptive n ... | 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;Turkey;Netherlands;Switzerland | ||
| 3 | NCT04659863 (ClinicalTrials.gov) | February 16, 2021 | 2/12/2020 | Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercho ... | 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) Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2] ... | Familial Hypercholesterolemia - Homozygous | Drug: Inclisiran;Drug: Placebo | Novartis Pharmaceuticals | NULL | Recruiting | 12 Years | 17 Years | All | 12 | Phase 3 | United States;Canada;France;Greece;Lebanon;Netherlands;Slovenia;Switzerland;Turkey |
| 4 | 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 ... | 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 ... | 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] Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 1005708 ... | Product Name: inclisiran sodium Product Code: KJX839 INN or Proposed INN: inclisiran Other descriptive name: Inclisiran sodium Product Name: inclisiransodium Product Code: KJX839 INN or Proposed INN: inclisiran Other descriptive n ... | 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 | ||
| 5 | 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 Study to evaluate efficacy and safety of inclisiran in adolescents with homozygous familial hypercho ... | 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 ... | 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] Homozygous Familial Hypercholesterolemia MedDRA version: 20.0;Level: LLT;Classification code 1005708 ... | Product Name: inclisiran sodium Product Code: KJX839 INN or Proposed INN: inclisiran Other descriptive name: Inclisiran sodium Product Name: inclisiransodium Product Code: KJX839 INN or Proposed INN: inclisiran Other descriptive n ... | 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 Serbia;United States;Slovenia;Greece;Lebanon;Turkey;Italy;Switzerland;France;Canada;South Africa;Ger ... | ||
| 6 | NCT04681170 (ClinicalTrials.gov) | November 23, 2020 | 7/12/2020 | 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 Phase III, Single Arm, Open Label, International, Multi Centre Study to Evaluate the Efficacy and Sa ... | 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 |
| 7 | 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 A clinical trial to assess the safety and effect of the drug lomitapide in the treatment of children ... | 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 Phase III, single-arm, open-label, international, multi-centre study to evaluate the efficacy and sa ... | 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] Homozygous familial hypercholesterolaemia (HoFH). A rare and life-threatening inherited disorder of ... | 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 Trade Name: Lojuxta5 mg hard capsules Product Name: Lojuxta5 mg hard capsules Product Code: [PRD725583 ... | 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 | ||
| 8 | 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 trial to evaluate the Efficacy and Safety of Evinacumab in Paediatric Patients With Homozygous Fam ... | A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics 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 ... | 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] Homozygous familial hypercholesterolemia MedDRA version: 21.0;Level: LLT;Classification code 1002060 ... | 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: ... | 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 United States;Taiwan;Ukraine;Lebanon;Austria;Italy;France;Canada;Jordan;Belgium;Australia;Netherland ... | ||
| 9 | 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 A Study to Determine the Safety, Tolerability, and Efficacy of SHR-1209 in Patients With Familial Hy ... | Open-label, Single-arm, Multicentre Study to Evaluate Efficacy and Safety of SHR-1209 in Subjects With Homozygous Familial Hypercholesterolemia Open-label, Single-arm, Multicentre Study to Evaluate Efficacy and Safety of SHR-1209 in Subjects Wi ... | Familial Hypercholesterolemia | Drug: SHR-1209 | Jiangsu HengRui Medicine Co., Ltd. | NULL | Not yet recruiting | 18 Years | 80 Years | All | 8 | Phase 2 | NULL |
| 10 | 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 An Open-label, Single-arm Study Evaluated the Efficacy and Safety of JS002 in Patients With Homozygo ... | Hyperlipemia | Drug: JS002 | Shanghai Junshi Bioscience Co., Ltd. | NULL | Active, not recruiting | 12 Years | 75 Years | All | 30 | Phase 2 | China |