A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
Completed
Conditions studied: Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
In brief
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.
Key facts
- Study ID
- NCT03597399
- Run by
- Spark Therapeutics, Inc.
- People needed
- 87
- Starts
- 2019-01-10
- Expected to finish
- 2025-06-30
- Last updated by the study team
- 2025-09-22
Who can join
Age: 1 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Received voretigene neparvovec-rzyl in at least one eye.
- Signed informed consent/assent (when applicable). These are obtained as required under institutional policies and applicable laws and regulations unless a consent waiver is obtained from the Institutional Review Board (IRB)/Independent Ethics Committee (IEC).
You may not qualify if…
- Previously participated in, or are currently participating in, a Spark Therapeutics clinical trial and received voretigene neparvovec-rzyl in both eyes.
Where it is running
- Children's Hospital of Los Angeles — Los Angeles, California, United States
- Bascom Palmer Eye Institute — Miami, Florida, United States
- University of Iowa Hospitals & Clinics — Iowa City, Iowa, United States
- Massachusetts Eye and Ear Institute — Boston, Massachusetts, United States
- Kellogg Eye Center — Ann Arbor, Michigan, United States
- Cincinnati Eye Institute — Cincinnati, Ohio, United States
- Casey Eye Institute — Portland, Oregon, United States
- Children's Hospital of Philadelphia (CHOP) — Philadelphia, Pennsylvania, United States
- Scheie Eye Institute — Philadelphia, Pennsylvania, United States
- Cullen Eye Institute — Houston, Texas, United States
Full record on ClinicalTrials.gov
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