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2 studies found for:    16474400 [PUBMED-IDS]
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Rank Status Study
1 Not yet recruiting Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
Conditions: Leber Congenital Amaurosis (LCA);   Eye Diseases;   Eye Diseases, Hereditary;   Retinal Diseases
Intervention: Genetic: AAV2/5-OPTIRPE65
2 Recruiting A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH)
Condition: Homozygous Familial Hypercholesterolemia
Intervention: Genetic: AAV directed hLDLR gene therapy

Study has passed its completion date and status has not been verified in more than two years.