79. 家族性高コレステロール血症（ホモ接合体）
［臨床試験数：128，薬物数：105（DrugBank：29），標的遺伝子数：8，標的パスウェイ数：15］
Searched query = "Homozygous familial hypercholesterolemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
| 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 | NCT04659863 (ClinicalTrials.gov) | January 29, 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) | Homozygous Familial Hypercholesterolemia | Drug: Inclisiran;Drug: Placebo | Novartis Pharmaceuticals | NULL | Not yet recruiting | 12 Years | 17 Years | All | 12 | Phase 3 | NULL |
| 2 | NCT03814187 (ClinicalTrials.gov) | April 16, 2019 | 17/1/2019 | Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C | An Open-label Extension Trial of the Phase III Lipid-lowering Trials to Assess the Effect of Long Term Dosing of Inclisiran Given as Subcutaneous Injections in Subjects With High Cardiovascular Risk and Elevated LDL-C | ASCVD;Elevated Cholesterol;Heterozygous Familial Hypercholesterolemia;Homozygous Familial Hypercholesterolemia | Drug: Inclisiran Sodium | Novartis | NULL | Active, not recruiting | 18 Years | N/A | All | 2991 | Phase 3 | United States;Canada;Czechia;Denmark;Germany;Hungary;Netherlands;Poland;South Africa;Spain;Sweden;Ukraine;United Kingdom |
| 3 | 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: Placebos | Novartis Pharmaceuticals | NULL | Active, not recruiting | 18 Years | 80 Years | All | 56 | Phase 3 | Hong Kong;Israel;Russian Federation;Serbia;South Africa;Taiwan;Turkey;Ukraine;Czechia |
| 4 | EUCTR2016-003376-49-NL (EUCTR) | 20/06/2017 | 18/01/2017 | A study to evaluate the effect of ALN-PCSSC treatment in patients with Homozygous Familial Hypercholesterolemia | An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects with Homozygous Familial Hypercholesterolemia - ORION-2 | Homozygous Familial Hypercholesterolemia MedDRA version: 19.1;Level: LLT;Classification code 10057080;Term: Homozygous familial hypercholesterolemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14] | Product Name: ALN-PCSSC Product Code: ALN-PCSSC INN or Proposed INN: ALN-60212 Other descriptive name: ALN-60212 | The Medicines Company | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United States;South Africa;Netherlands | ||
| 5 | NCT02963311 (ClinicalTrials.gov) | December 13, 2016 | 10/11/2016 | A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) | An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH) | Homozygous Familial Hypercholesterolemia | Drug: ALN-PCSSC;Drug: Standard of Care | The Medicines Company | NULL | Completed | 12 Years | N/A | All | 9 | Phase 2 | United States;Netherlands;South Africa |
| 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 |
| 6 | EUCTR2020-002755-38-NL (EUCTR) | 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 | NA | Female: yes Male: yes | 12 | Phase 3 | France;United States;Serbia;Slovenia;Greece;Canada;Lebanon;Turkey;Netherlands;Switzerland |