drugset
drugset / Trial / NCT02064569

Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients

NCT02064569 ↗

Phase 1/2 Completed 19 enrolled GenSight Biologics
NaSingle-groupOpen-labelTreatment

Summary

The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.

Timeline

Start
2014-02-13
Primary completion
2020-06-25
Completion
2020-06-25

Drugs

EvaluationDrugModalityDoseRoute
Subject GS010 Gene therapy (AAV / viral vector) — —

Indications

Leber hereditary optic neuropathy