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Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

NCT00749957 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2017-12-28

Study results available
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Summary

The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene.

Funding Source - FDA OOPD

Conditions

  • Leber Congenital Amaurosis

Interventions

BIOLOGICAL

rAAV2-CB-hRPE65

Recombinant adeno-associated virus vector expressing RPE65

Sponsors & Collaborators

  • Oregon Health and Science University

    collaborator OTHER

  • University of Massachusetts, Worcester

    collaborator OTHER

  • Beacon Therapeutics

    lead INDUSTRY

Principal Investigators

  • J Timothy Stout, MD, PhD, MBA · Casey Eye Institute, Oregon Health & Science University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-06-17
Primary Completion
2014-09-23
Completion
2017-09-22

Countries

  • United States

Study Locations

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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 NCT00749957 on ClinicalTrials.gov