drugset
drugset / Trial / NCT01496040

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

NCT01496040 ↗

Phase 1/2 Completed 9 enrolled Nantes University Hospital
NaSingle-groupOpen-label

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.

Timeline

Start
2011-09
Primary completion
2014-08
Completion
2014-08

Drugs

EvaluationDrugModalityDoseRoute
Subject rAAV2/4.hRPE65 Gene therapy (AAV / viral vector) 0.4 ml Other
Subject rAAV2/4.hRPE65 Gene therapy (AAV / viral vector) 0.8 ml Other

Indications

Leber congenital amaurosis