NCT01496040

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

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

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Nantes University Hospital · Academic / Other
• Sex: All
• Age: 6 Years – 50 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
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.

Timeline

• Start date: 2011-09-01
• Primary completion: 2014-08-01
• Completion: 2014-08-01
• First posted: 2011-12-21
• Last updated: 2015-10-07

Locations

1 site across 1 country: France