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Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCT00999609 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 31

Last updated 2025-04-23

Study results available
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Summary

The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl). At least twenty-four subjects, three years of age or older, will be recruited. The intervention group will receive AAV2-hRPE65v2 at either The Children's Hospital of Philadelphia or University of Iowa to determine if it improves visual and retinal function in individuals with RPE65 gene mutations.

Conditions

  • Inherited Retinal Dystrophy Due to RPE65 Mutations
  • Leber Congenital Amaurosis

Interventions

BIOLOGICAL

AAV2-hRPE65v2,voretigene neparvovec-rzyl

Subretinal administration of gene therapy vector AAV2-hRPE65v2 (1.5E11 vector genomes per eye) to both eyes via surgical procedures on separate days.

Sponsors & Collaborators

Principal Investigators

  • Albert M Maguire, MD · Children's Hospital of Philadelphia

  • Stephen R Russell, MD · University of Iowa

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-10-31
Primary Completion
2015-07-31
Completion
2030-01-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

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 NCT00999609 on ClinicalTrials.gov