NCT02693119·Phase 2·INTERVENTIONAL

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

Elamipretide Pain Completed

Status

Completed

Phase

Phase 2

Enrollment

Locations

Results

Posted

Publications

Study summary

What the protocol is testing.

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.

Full detailed description

This was a prospective, randomized, double-masked (DM), vehicle-controlled, single-center study plus open label extension period (OLE) in which approximately 12 subjects with LHON having the genetic mtDNA mutation m.11778G\>A were randomized in a masked manner into 1 of 3 groups in a 1:1:1 ratio: one drop of elamipretide 1.0% topical ophthalmic solution twice daily (BID) in the: left eye, right eye, or both eyes. After completion of the 52-week treatment period or the Week 56 follow-up period, subjects were invited to participate in the OLE period for up to 108 weeks. During the OLE participants received 1% topical opthalmic elamipretide in both eyes (OU) daily. If a subject did not consent to the OLE, he/she completed the study at the Week 56 (±7 days) visit. There were 4 periods in this study: (1) screening period (up to 6 weeks); (2) double-masked treatment period (52 weeks); (3) OLE period (up to 108 weeks), and (4) follow-up period (4 weeks) after completion of End-of-Treatment (EOT) visit.

Interventions

Treatment arms and agents.

DRUG

elamipretide (MTP-131) 1% topical ophthalmic solution

DRUG

Vehicle topical ophthalmic solution

Timeline

From registration to results.

First posted
Feb 26, 2016
Study start
Mar 2016
Primary completion
Sep 2019
Study completion
Dec 2019
Results posted
Oct 12, 2021
Registry updated
Nov 30, 2021

Outcomes

What the study measures.

Primary outcomes

Double Masked Period: Incidence of Ocular TEAEs

Time frame · 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)

The incidence of ocular treatment emergent adverse events (TEAEs).

Open Label Extension: Incidence of Ocular TEAEs

Time frame · Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)

Open Label Extension: The incidence of ocular treatment emergent adverse events (TEAEs).

Double Masked Period: Severity of Ocular TEAEs

Time frame · 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)

The severity of ocular treatment emergent adverse events (TEAEs).

OLE: Severity of Ocular TEAEs

Time frame · Assessed at Week 68, 84, 100, 116, 132, 148, 152, Week 160/ (End of treatment visit)

The severity of ocular treatment emergent adverse events (TEAEs).

Double Masked Period: Best Corrected Visual Acuity (BCVA)

Time frame · 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)

Double Masked Period: Best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale by visit. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome).

Open Label Extension Period: Best Corrected Visual Acuity (BCVA) Using the Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale

Time frame · Assessed at each visit from Baseline from Week 68 to Week 160 (follow-up visit).

Open Label Extension Period: Best corrected visual acuity (BCVA) using the using the Early Treatment Diabetic Retinopathy Study (ETDRS) scale by visit. ETDRS charts present a series of five letters of equal difficulty on each row, with standardized spacing between letters and rows; there is a total of 14 lines (70 letters), with letter size increasing further geometrically and equivalently in every line by a factor of 1.2589 (or 0.1 log unit), moving up the chart. Minimum score of zero, maximum score of 100. Change from baseline: a more negative score is worse outcome, a more positive score is better outcome. A lower score means less letters were read correctly (worse outcome) and a higher score means more letters were read correctly (better outcome).