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Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

NCT03602820 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 41

Last updated 2025-04-18

No results posted yet for this study

Summary

Multi-site, non-randomized, observational study, for up to 15 years after subretinal AAV2-hRPE65v2 administration for each subject. The study is a non-interventional, follow-up study of subjects who participated in previous AAV2-hRPE65v2 gene therapy clinical trials.

Conditions

  • Inherited Retinal Dystrophy Due to RPE65 Mutations

Interventions

BIOLOGICAL

AAV2-hRPE65v2

Sponsors & Collaborators

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Albert Maguire, MD · University of Pennsylvania

  • Stephen Russell, MD · University of Iowa

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-06-30
Primary Completion
2030-01-31
Completion
2030-01-31
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03602820 on ClinicalTrials.gov