NCT03597399Completed
A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec in US
Source: ClinicalTrials.gov · Last updated 22 Sept 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Study type
- Observational
- Enrollment
- 87 (actual)
- First submitted
- 12 Jul 2018
- Start date
- 10 Jan 2019
- Primary completion date
- 30 Jun 2025
- Lead sponsor
- Spark Therapeutics, Inc.
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT03597399
What you commit to
- Study type: Observational
- Participants enrolled: 87
- From 10 Jan 2019 to the primary completion date, 30 Jun 2025
- 10 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 1\. Previously participated in, or are currently participating in, a Spark Therapeutics clinical trial and received voretigene neparvovec-rzyl in both eyes.
Who the study is looking for
- 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).
Age in the registry: 12 months to - · Sex: All · Does not accept healthy volunteers
Sites (10)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Kellogg Eye Center Coordinator of this site? Confirm its status | Ann Arbor | United States | Unknown status |
| Massachusetts Eye and Ear Institute Coordinator of this site? Confirm its status | Boston | United States | Unknown status |
| Cincinnati Eye Institute Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
| Cullen Eye Institute Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| University of Iowa Hospitals & Clinics Coordinator of this site? Confirm its status | Iowa City | United States | Unknown status |
| Children's Hospital of Los Angeles Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Bascom Palmer Eye Institute Coordinator of this site? Confirm its status | Miami | United States | Unknown status |
| Children's Hospital of Philadelphia (CHOP) Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Scheie Eye Institute Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Casey Eye Institute Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.