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Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65

NCT01496040 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2015-10-07

No results posted yet for this study

Summary

The purpose of the study is to assess the safety and efficacy of the active substance rAAV-2/4.hRPE65 in patients with Leber Congenital Amaurosis or Congenital severe early-onset retinal degeneration associated with RPE65 mutation.

Conditions

  • Leber Congenital Amaurosis

Interventions

DRUG

rAAV2/4.hRPE65

One injection in on eye Cohorte 1 : 3 patients will receive one injection of up to 400 microliters of the IMP Cohorte 2 : 3 patients will receive one injection of up to 800 microliters of the IMP. Cohorte 3 : 3 patients under age of eighteen will receive one injection up to 400 or 800 microliters of the IMP.

Sponsors & Collaborators

  • Nantes University Hospital

    lead OTHER

Principal Investigators

  • Michel WEBER, Professor · Nantes University Hospital

Study Design

Allocation
NA
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-09-30
Primary Completion
2014-08-31
Completion
2014-08-31

Countries

  • France

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 NCT01496040 on ClinicalTrials.gov