79. Homozygous familial hypercholesterolemia Clinical trials / Disease details
Clinical trials : 145 / Drugs : 114 - (DrugBank : 26) / Drug target genes : 8 - Drug target pathways : 17
Showing 1 to 10 of 145 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 | 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 ... | Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia in a Real Life Setting in Canada Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia ... | 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) Phase 2 Study to Evaluate the Safety and Efficacy of ARO-ANG3 in Subjects With Homozygous Familial H ... | 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. 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 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] Patients with elevated LDL-C, with homozygous familial hypercholesterolemia(HoFH) and heterozygous f ... | 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 | 2000 | Phase 3 | United States;France;Canada;Spain;Turkey;South Africa;Israel;Norway;Germany;United Kingdom;New Zealand;India United States;France;Canada;Spain;Turkey;South Africa;Israel;Norway;Germany;United Kingdom;New Zeala ... | ||
| 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. 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 ... | ||
| 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 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 ... | 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 Trade Name: Leqvio284 mg solution for injection in pre-filled syringe Product Name: inclisiransodium P ... | 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 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 | 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 ... | 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 | ||
| 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 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 ... | ||
| 9 | NCT04681170 (ClinicalTrials.gov) | December 14, 2020 | 7/12/2020 | Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolae ... | 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 |
| 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 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 |