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Completed NCT01496040

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

Conditions: Leber Congenital Amaurosis

Sex: All

Ages: 6 Years – 50 Years

Healthy volunteers: No

Phase: PHASE1, PHASE2

Enrollment: 9

Sponsor: Nantes University Hospital

Location: CHU Nantes Nantes

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.

Eligibility 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 \

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT01496040). StuddyBuddy aggregates publicly available trial information.