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Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

NCT00643747 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2015-12-07

No results posted yet for this study

Summary

The purpose of the study is to determine whether gene therapy is safe and effective for the treatment of severe childhood blindness caused by mutations in RPE65.

Conditions

  • Retinal Degeneration

Interventions

BIOLOGICAL

tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)

Single subretinal injection of vector suspension; up to 3x10e12 vector particles

Sponsors & Collaborators

  • Moorfields Eye Hospital NHS Foundation Trust

    collaborator OTHER

  • Targeted Genetics Corporation

    collaborator INDUSTRY

  • University College, London

    lead OTHER

Principal Investigators

  • Robin R Ali, PhD · University College, London

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
5 Years
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-01-31
Primary Completion
2014-12-31
Completion
2014-12-31

Countries

  • United Kingdom

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 NCT00643747 on ClinicalTrials.gov