MedTrace Logo

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

NCT03597399 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 87

Last updated 2025-09-22

No results posted yet for this study

Summary

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.

Conditions

  • Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy

Interventions

BIOLOGICAL

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.

Sponsors & Collaborators

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Clinical Ophthalmic Lead · Spark Therapeutics, Inc.

Eligibility

Min Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-10
Primary Completion
2025-06-30
Completion
2025-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

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 NCT03597399 on ClinicalTrials.gov