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 53 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 | EUCTR2020-002755-38-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 | ||
| 2 | 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 |
| 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] ... | 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 | ||
| 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 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 ... | ||
| 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) A Two-Part (Double-Blind PlaceboControlled/Open-Label) Multicenter Study to Evaluate Safety, Tolerab ... | 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 STUDY TO EVALUATE THE EFFICACY AND SAFETY OF EVINACUMAB IN PATIENTS WITH HOMOZYGOUS FAMILIAL HYPERCH ... | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLEL-GROUP 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 SA ... | 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 Intervention name : Evinacumab INN of the intervention : Evinacumab Dosage And administration of the ... | 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 study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high ... | A randomized, double-blind, placebo-controlled, parallel-group 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 sa ... | 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] Homozygous familial hypercholesterolemia MedDRA version: 20.1;Level: LLT;Classification code 1002060 ... | 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 Product Name: Evinacumab(REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descrip ... | 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 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Africa;Germany;Netherlands United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Afric ... | ||
| 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 study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high ... | A randomized, double-blind, placebo-controlled, parallel-group 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 sa ... | 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 ... | 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 Trade Name: Praluent Product Name: Alirocumab Product Code: [REGN7272] INN or Proposed INN: Alirocumab O ... | 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 United States;Czechia;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;Sou ... | ||
| 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 study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high ... | A randomized, double-blind, placebo-controlled, parallel-group 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 sa ... | 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 (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 Product Name: Evinacumab(REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descrip ... | 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 United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Afric ... | ||
| 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 study to evaluate the safety and efficacy of evinacumab in patients with Hereditary abnormal (high ... | A randomized, double-blind, placebo-controlled, parallel-group 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 sa ... | 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] Homozygous familial hypercholesterolemia MedDRA version: 20.1;Level: LLT;Classification code 1002060 ... | 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 Product Name: Evinacumab(REGN1500) Product Code: REGN1500 INN or Proposed INN: Evinacumab Other descrip ... | 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 United States;Greece;Ukraine;Turkey;Austria;Italy;France;Czech Republic;Canada;Australia;South Afric ... |