A Phase 1/2, Single-Arm, Open-Label, Dose-Finding Clinical Trial to Evaluate the Safety and Efficacy of Gene Therapy for Leber's Hereditary Optic Neuropathy (LHON) Associated with ND4 Mutation
Adult
- Age at the time of signing the informed consent form: the age of the subjects must be ≥18 years old and ≤75 years old - The clinical manifested vision loss due to LHON, and any eye BCVA ≥0.5 LogMAR - The genotype testing result shows the presence of G11778A mutation in the ND4 gene, and the absence of the other primary LHON associated mutations in the mitochondrial DNA (mtDNA) (ND1 [G3460A] or ND6 [T14484C]) (confirmed by a CLIA-certified international laboratory) - The vision loss in the eye with worse visual acuity lasted >6 months and <10 years at screening - Pupils can be adequately dilated for a thorough ocular examination and visual acuity test - Each eye of the subject must maintain at least Hand Motion visual acuity (VA) (≤2.3 LogMAR) as defined in the ocular/vision examination manual (operating manual for refraction and VA examinations) in this study - Willingness to comply with the clinical study protocol and 5 years of long-term follow-up after administration
Treatment
University of Colorado Hospital
Prem Subramanian, MD, PhD
Protocol Number: 22-2250
More information available at ClinicalTrials.gov: NCT05293626
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