Clinical Trial: A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide (MTP-131) Topical Ophthalmic Solution for the Treatment of Leber's Hereditary Optic Neuropathy
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Prospective, Randomized, Double-Masked, Vehicle Controlled, Phase 2 Clinical Study to Evaluate the Safety, Tolerability and Efficacy of Elamipretide (MTP-131) Topical Ophthalmic Solution in Subjects
Brief Summary: 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.
Detailed Summary:
Sponsor: Stealth BioTherapeutics Inc.
Current Primary Outcome: Incidence and severity of adverse events [ Time Frame: Assessed at each visit from Baseline to Week 56 (follow-up visit) ]
Original Primary Outcome: Incidence and severity of adverse events [ Time Frame: Assessed at each visit from Baseline to Week 16 (follow-up visit) (total 7 visits) ]
Current Secondary Outcome:
- Change in photopic negative response electroretinography (PhNR-ERG) response pattern [ Time Frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. ]
- Change in visual field MD as measured by Humphrey automated visual field testing stimulus III [ Time Frame: Assessed at each visit from Baseline to Week 56 (follow-up visit), except for Day 5 visit. ]
- Change in best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale [ Time Frame: Assessed at each visit from Baseline to Week 56 (follow-up visit). ]
- Change in Visual Function Questionnaire (VFQ-39) score [ Time Frame: Assessed at Baseline, Week 36 (end-of-treatment visit) and Week 56 (follow-up visit) ]
- Change in mean retinal nerve fiber layer thickness by spectral domain optical coherence tomography (SD-OCT) [ Time Frame: Assessed at Baseline and Week 52 (end-of-treatment visit) ]
- Change in mean retinal ganglion cell layer thickness by spectral domain optical coherence tomography (SD-OCT) [ Time Frame: Assessed at Baseline and Week 52 (end-of-treatment visit) ]
Original Secondary Outcome:
- Change in photopic negative response electroretinography (PhNR-ERG) response pattern [ Time Frame: Assessed at each visit from Baseline to Week 16 (follow-up visit), except for Day 5 visit (total 6 visits). ]
- Change in visual field MD as measured by Humphrey automated visual field testing stimulus III [ Time Frame: Assessed at each visit from Baseline to Week 16 (follow-up visit), except for Day 5 visit (total 6 visits). ]
- Change in best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale [ Time Frame: Assessed at each visit from Baseline to Week 16 (follow-up visit) (total 7 visits). ]
- Change in Visual Function Questionnaire (VFQ-39) score [ Time Frame: Assessed at Baseline, Week 12 (end-of-treatment visit) and Week 16 (follow-up visit) ]
- Change in mean retinal nerve fiber layer thickness by spectral domain optical coherence tomography (SD-OCT) [ Time Frame: Assessed at Baseline and Week 12 (end-of-treatment visit) ]
- Change in mean retinal ganglion cell layer thickness by spectral domain optical coherence tomography (SD-OCT) [ Time Frame: Assessed at Baseline and Week 12 (end-of-treatment visit) ]
Information By: Stealth BioTherapeutics Inc.
Dates:
Date Received: February 15, 2016
Date Started: March 2016
Date Completion: May 2018
Last Updated: May 4, 2017
Last Verified: May 2017