Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCT00999609 · clinicaltrials.gov ↗

PHASE3

Phase

ACTIVE_NOT_RECRUITING

Status

31

Enrollment

INDUSTRY

Sponsor class

Conditions

Inherited Retinal Dystrophy Due to RPE65 Mutations
Leber Congenital Amaurosis

Interventions

BIOLOGICAL: AAV2-hRPE65v2,voretigene neparvovec-rzyl

Sponsor

Spark Therapeutics, Inc.

Collaborators

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