NCT03597399View on ClinicalTrials.gov

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

CompletedOBSERVATIONAL
Enrollment

87

Participants

Timeline

Start Date

January 10, 2019

Primary Completion Date

June 30, 2025

Study Completion Date

June 30, 2025

Conditions
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Interventions
BIOLOGICAL

AAV2-hRPE65v2,voretigene neparvovec-rzyl

Subretinal administration of gene therapy vector AAV2-hRPE65v2 (voretigene neparvovec-rzyl) to both eyes via surgical procedures on separate days.

Trial Locations (10)

19104

Children's Hospital of Philadelphia (CHOP), Philadelphia

Scheie Eye Institute, Philadelphia

33136

Bascom Palmer Eye Institute, Miami

45242

Cincinnati Eye Institute, Cincinnati

48105

Kellogg Eye Center, Ann Arbor

52242

University of Iowa Hospitals & Clinics, Iowa City

77030

Cullen Eye Institute, Houston

90027

Children's Hospital of Los Angeles, Los Angeles

97239

Casey Eye Institute, Portland

02114

Massachusetts Eye and Ear Institute, Boston

All Listed Sponsors
lead

Spark Therapeutics, Inc.

INDUSTRY

NCT03597399 - A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US | Biotech Hunter | Biotech Hunter