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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
6 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1NCT04659863
(ClinicalTrials.gov)
January 29, 20212/12/2020Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial HypercholesterolemiaTwo 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 HypercholesterolemiaDrug: Inclisiran;Drug: PlaceboNovartis PharmaceuticalsNULLNot yet recruiting12 Years17 YearsAll12Phase 3NULL
2NCT03814187
(ClinicalTrials.gov)
April 16, 201917/1/2019Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-CAn 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-CASCVD;Elevated Cholesterol;Heterozygous Familial Hypercholesterolemia;Homozygous Familial HypercholesterolemiaDrug: Inclisiran SodiumNovartisNULLActive, not recruiting18 YearsN/AAll2991Phase 3United States;Canada;Czechia;Denmark;Germany;Hungary;Netherlands;Poland;South Africa;Spain;Sweden;Ukraine;United Kingdom
3NCT03851705
(ClinicalTrials.gov)
February 6, 20197/2/2019A 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 HypercholesterolemiaDrug: Inclisiran for injection;Drug: PlacebosNovartis PharmaceuticalsNULLActive, not recruiting18 Years80 YearsAll56Phase 3Hong Kong;Israel;Russian Federation;Serbia;South Africa;Taiwan;Turkey;Ukraine;Czechia
4EUCTR2016-003376-49-NL
(EUCTR)
20/06/201718/01/2017A study to evaluate the effect of ALN-PCSSC treatment in patients with Homozygous Familial HypercholesterolemiaAn 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 CompanyNULLNot RecruitingFemale: yes
Male: yes
10Phase 2United States;South Africa;Netherlands
5NCT02963311
(ClinicalTrials.gov)
December 13, 201610/11/2016A 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 HypercholesterolemiaDrug: ALN-PCSSC;Drug: Standard of CareThe Medicines CompanyNULLCompleted12 YearsN/AAll9Phase 2United States;Netherlands;South Africa
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
6EUCTR2020-002755-38-NL
(EUCTR)
26/12/2020Study to evaluate efficacy and safety of inclisiran in adolescents with homozygous familial hypercholesterolemiaTwo 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 AGNULLNAFemale: yes
Male: yes
12Phase 3France;United States;Serbia;Slovenia;Greece;Canada;Lebanon;Turkey;Netherlands;Switzerland