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

• Status: Completed
• Trial end date: 2014-08-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Nantes University Hospital

Criteria

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........