Clinical trial
A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec in US
Name
SPKRPE-PASS
Description
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.
Trial arms
Trial start
2019-01-10
Estimated PCD
2025-06-01
Trial end
2025-06-01
Status
Active (not recruiting)
Treatment
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.
Other names:
AAV2-hRPE65v2, voretigene neparvovec, gene therapy vector
Size
87
Primary endpoint
Collection of all Adverse Events and Serious Adverse Events
up to 5 years
Eligibility criteria
Inclusion Criteria:
1. Received voretigene neparvovec-rzyl in at least one eye.
2. 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).
Exclusion Criteria:
1. Previously participated in, or are currently participating in, a Spark Therapeutics clinical trial and received voretigene neparvovec-rzyl in both eyes.
Protocol
{'studyType': 'OBSERVATIONAL', 'patientRegistry': True, 'targetDuration': '5 Years', 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'OTHER'}, 'enrollmentInfo': {'count': 87, 'type': 'ACTUAL'}}
Updated at
2024-01-23
1 organization
4 products
1 indication
Organization
Spark TherapeuticsProduct
AAV2-hRPE65v2Product
voretigene neparvovec-rzyl