A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US
NCT ID: NCT03597399
Last Updated: 2025-09-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
87 participants
OBSERVATIONAL
2019-01-10
2025-06-30
Brief Summary
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The enrollment period will last for two years from the first treatment following product approval (through 31March2020) and include a minimum of 40 patients.
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Detailed Description
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This post authorization safety study will focus on further characterizing the long-term safety profile of voretigene neparvovec-rzyl in patients with RPE65 mutation-associated retinal dystrophy using an observational, longitudinal design.
Conditions
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Study Design
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COHORT
OTHER
Interventions
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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 Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
12 Months
ALL
No
Sponsors
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Spark Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Ophthalmic Lead
Role: STUDY_DIRECTOR
Spark Therapeutics, Inc.
Locations
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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
Countries
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Other Identifiers
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SPKRPE-PASS
Identifier Type: -
Identifier Source: org_study_id
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