This is a Phase 2, prospective, randomized, double-masked, vehicle controlled, single-center study in approximately 12 subjects with LHON to evaluate safety, tolerability and efficacy of elamipretide (MTP-131) topical ophthalmic solution in this patient population. At the conclusion of 52 weeks of treatment, subjects will be offered the opportunity to enter an Open Label Extension for up to 48 additional weeks of treatment.
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Double Masked Period: Incidence of Ocular TEAEs
Timeframe: Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit)
Open Label Extension: Incidence of Ocular TEAEs
Timeframe: Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)
Double Masked Period: Severity of Ocular TEAEs
Timeframe: Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit)
OLE: Severity of Ocular TEAEs
Timeframe: Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)
Double Masked Period: Best Corrected Visual Acuity (BCVA)
Timeframe: Assessed at each visit: Baseline, Day 5 Visit, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 (End of treatment visit) and Week 56 (Follow up visit)
Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale
Timeframe: Assessed at each visit from Baseline from Week 68 to Week 160 (follow-up visit).