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

October 6, 2015 updated by: Nantes University Hospital

Prospective Monocentric Open Label Non Randomized Uncontrolled Phase I/II Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

9

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Nantes, France, 44000
        • CHU Nantes

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 50 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Mutations that code for abnormal RPE65 protein
  • Presence of characteristic abnormalities in fundus
  • Dramatic reduction of both rods ans cones ERG responses
  • Low visual acuity <0.32
  • inform consent signed

Exclusion Criteria:

  • Patients with chronic conditions such a haematological, cardiac, renal diseases
  • Patients with, within the past 6 months, a clinically significant cardiac disease or known congestive heart failure, cardiac rhytm and conduction abnormalities
  • Patients with pulmonaty dysfunction
  • Patients with suspected rheumatoid arthritis
  • Patients with current systemic infection........

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: rAAV2/4.hRPE65

3 cohortes of 3 patients each.

All the patients enrolled in the study will receive a single subretinal injection in one eye. The eye, that will be injected, will be the eye with the poorest visual acuity.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The drug safety evaluation after administration
Time Frame: After administration of the gene therapy product.The patient will be folloed for the duration of the hospital stay, an average of 7 days
Biodistribution : Urine sampling and nasal secretion will be collected at several time points after administration of the gene therapy product during all the duration of hospital stay, an average of 7 days.
After administration of the gene therapy product.The patient will be folloed for the duration of the hospital stay, an average of 7 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Different efficacy parameters and immune parameters have to be measured to conclude on the overall amelioration of quality of life of enrolled patients
Time Frame: Between Day -120 and Day-7, Day 5, Day 14, Day 30 Day 60, Day 90, Day 120, Day 180, Day 360

Recording global ERG (electroretinogram)

Patient efficacy questionnaire

Testing of far and near visual acuity, color vision, pupillometry, microperimetry and dark adaptation.
Between Day -120 and Day-7, Day 5, Day 14, Day 30 Day 60, Day 90, Day 120, Day 180, Day 360

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nantes University Hospital

Investigators

  • Principal Investigator: Michel WEBER, Professor, CHU Nantes

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2011

Primary Completion (Actual)

August 1, 2014

Study Completion (Actual)

August 1, 2014

Study Registration Dates

First Submitted

November 24, 2011

First Submitted That Met QC Criteria

December 16, 2011

First Posted (Estimate)

December 21, 2011

Study Record Updates

Last Update Posted (Estimate)

October 7, 2015

Last Update Submitted That Met QC Criteria

October 6, 2015

Last Verified

October 1, 2015

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BRD 07/08-K
  • 2011-000418-21 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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