A Phase 1/2, Multi-regional, Single-Arm, Open-Label, Dose-Finding Clinical Trial to Evaluate the Safety, Tolerability and Efficacy of Gene Therapy for Leber's Hereditary Optic Neuropathy (LHON) Associated with ND1 Mutation
The study drug is an investigational gene therapy product. Gene therapy is a technique that uses genes to treat or cure disease. “Investigational” means that the study drug is currently being tested and is not approved by the Food and Drug Administration (FDA) for sale in the United States. The study drug contains a copy of the working ND1 gene. It is hoped that the study drug will make up for the faulty ND1 gene, and mediate normal ND1 protein expression in the mitochondria of the eye causing the vision loss in LHON.
MoreAdult
- you are between 18 and 75 years of age - you have vision loss due to Leber’s Hereditary Optic Neuropathy Associated with ND1 Mutation
Treatment
University of Colorado Hospital
Prem Subramanian, MD, PhD
Protocol Number: 23-0981
More information available at ClinicalTrials.gov: NCT05820152
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