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.

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1NCT02963311December 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