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

What Was Tested

The trial (phase 2) is built around SS-31 (Elamipretide).

It had four arms (groups): Group 1 (experimental), Group 2 (experimental), Group 3 (experimental) and OLE (experimental).

The registry classes the interventions as drug.

More on the Plan

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.

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.

Who Took Part

The registry lists 12 actual participants took part.

It was open to people of any sex aged from 18 years up to 50 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Adults ≥18 and ≤ 50 years old at the time of loss of vision in the second eye. Able to provide informed consent and willing to comply with all study visits and examinations. Loss of vision in both eyes of ≥1 year and ≤10 years at the time of the Screening Visit and current clinically stable visual function (as assessed by the Investigator). Documentation of having satisfactorily completed at least two previous Humphrey automated visual field tests prior to screening. Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the informed consent form.

The full record lists 7 inclusion and 7 exclusion criteria; among the exclusions: any other ocular pathology requiring treatment with prescription topical ophthalmic drops (e.g., glaucoma, dry eye); cup to disc ratio of \> 0.8 in either eye; media opacity, suboptimal pupillary dilatation, or refractive error that interferes with adequate retinal imaging.

How the Study Was Run

It was run as a randomized, parallel-group and quadruple-blind study.

Random assignment meant chance, not the researchers, decided who got SS-31 (Elamipretide) and who got the comparison, which limits bias.

The SS-31 (Elamipretide) group and the comparison group ran side by side rather than switching.

Blinding on both sides kept the participants and researchers unaware of who received SS-31 (Elamipretide).

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2016-03 with completion listed for 2019-09.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Double Masked Period: Incidence of Ocular 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)). Double Masked Period: Severity of Ocular TEAEs.

It also tracked 53 secondary outcomes, for example Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude, Open Label Extension Period: Photopic Negative Response Electroretinography (PhNR-ERG) A-wave Amplitude, Double Masked Period: Photopic Negative Response Electroretinography (PhNR-ERG) B-wave Amplitude, Open Label Extension Period: PhNR-ERG B-wave Amplitude, Double Masked Period: PhNR Amplitude and Open Label Extension Period: PhNR Amplitude.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of SS-31 (Elamipretide) in 12 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the SS-31 (Elamipretide) compound page, all human trials.