Trials / NCT03597399
A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
Completed Observational Natural history / observational
- Sponsor
- Spark Therapeutics, Inc.
- Treatment
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Gene
- RPE65
- Conditions
- Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
- Ages
- 12 Months to no upper limit
- Enrollment
- 87 participants
- Started
- 2019-01-10
- Main results expected
- 2025-06-30
- Results posted
- Not yet
- Record last updated
- 2025-09-22
What the study is testing
The objective of this study is to collect long-term safety information (i.e., for 5 years after treatment) associated with voretigene neparvovec-rzyl (vector and/or transgene), its subretinal injection procedure, the concomitant use of corticosteroids, or a combination of these procedures and products.
The enrollment period will last for two years from the first treatment following product approval (through 31March2020) and include a minimum of 40 patients.
Where it runs
United States (10)
- Children's Hospital of Los Angeles, Los Angeles, California
- Bascom Palmer Eye Institute, Miami, Florida
- University of Iowa Hospitals & Clinics, Iowa City, Iowa
- Massachusetts Eye and Ear Institute, Boston, Massachusetts
- Kellogg Eye Center, Ann Arbor, Michigan
- Cincinnati Eye Institute, Cincinnati, Ohio
- Casey Eye Institute, Portland, Oregon
- Children's Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania
- Scheie Eye Institute, Philadelphia, Pennsylvania
- Cullen Eye Institute, Houston, Texas