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
| 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 | EUCTR2020-002755-38-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] | 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;Turkey;Netherlands;Switzerland | ||
| 2 | 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 | Recruiting | 12 Years | 17 Years | All | 12 | Phase 3 | United States;Canada;France;Greece;Lebanon;Netherlands;Slovenia;Switzerland;Turkey |
| 3 | 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 | ||
| 4 | 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 | ||
| 5 | 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 for injection;Drug: Placebo | Novartis Pharmaceuticals | NULL | Completed | 18 Years | 80 Years | All | 56 | Phase 3 | Hong Kong;Israel;Russian Federation;Serbia;South Africa;Taiwan;Turkey;Ukraine;Czechia |
| 6 | 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 | |
| 7 | 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 | ||
| 8 | 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 | ||
| 9 | 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 | ||
| 10 | 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 | ||
| 11 | 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 |
| 12 | 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 | ||
| 13 | 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 | ||
| 14 | 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 | ||
| 15 | 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 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Czech Republic;Greece;Canada;Ukraine;Turkey;Austria;South Africa;Germany;Italy | ||
| 16 | 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 |
| 17 | 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 | ||
| 18 | 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 | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 61 | Phase 1;Phase 2 | Canada;Netherlands;Italy | ||
| 19 | 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 |
| 20 | 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 | ||
| 21 | 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 | ||
| 22 | 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 |
| 23 | 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 | ||
| 24 | 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 | ||
| 25 | 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 | ||
| 26 | 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 | |||
| 27 | 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 | |||
| 28 | 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 |
| 29 | 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 | ||
| 30 | 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 | |||
| 31 | 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 | |||
| 32 | 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 | ||
| 33 | 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 | ||
| 34 | 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 | ||
| 35 | 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 | |||
| 36 | 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 | ||
| 37 | 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 |
| 38 | 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 | ||
| 39 | 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 | ||
| 40 | 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 | |||
| 41 | 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 |
| 42 | 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 | ||
| 43 | 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 | ||
| 44 | 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 |
| 45 | 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 | |||
| 46 | 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 |
| 47 | 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 |
| 48 | 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 |
| 49 | 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 | |||
| 50 | 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 | |||
| 51 | 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 | |||
| 52 | 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 | |||
| 53 | 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 |