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

  
1 trial found
No.TrialIDDate_
enrollment
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agemin
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PhaseCountries
1NCT04080050
(ClinicalTrials.gov)
September 30, 20193/9/2019A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501Homozygous Familial Hypercholesterolemia (HoFH)Drug: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene TherapyRegenxbio Inc.NULLActive, not recruiting18 YearsN/AAll8United States;Canada;Netherlands;Italy