A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)

What Was Tested

In this phase 1, phase 2 trial, researchers set out to test SS-31 (Elamipretide).

It had four arms (groups): one (experimental), one (experimental), Placebo A (placebo comparator) and Part B Placebo (placebo comparator).

Part A: Each subject will receive one drop of vehicle solution BID in the paired eye of the randomly selected study eye (Cohort 1).

The registry classes the interventions as drug.

More on the Plan

This is a Phase 1/2 trial in two parts.

Who Took Part

The registry lists 22 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

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

Examples of who could join: Adults ≥18 years old at the time of Screening Visit. Diagnosis of FCED OU (both eyes) based on clinical and ophthalmic test findings. Clinical evidence of corneal edema OU diagnosed with FCED, including one or more of the following signs: corneal epithelial microcysts, corneal epithelial bullae, stromal folds, or stromal haze. Best-corrected distance visual acuity (BCVA) of 20/25 to 20/320 (inclusive) at the time of Screening Visit and Baseline Visit OU. Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the informed consent form (ICF) until after the last study.

The full record lists 8 inclusion and 10 exclusion criteria; among the exclusions: any ocular pathology requiring treatment with topical ophthalmic drops, with the exception of glaucoma or ocular hypertension; use of topical hypertonic saline drops for 3 days prior to screening and throughout the duration of the study; history of corneal disease (other than fced) or corneal surgery in either eye.

How the Study Was Run

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

Because people in this SS-31 (Elamipretide) study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Groups ran in parallel: each of the participants stayed in one arm throughout.

As a double-blind SS-31 (Elamipretide) study, the assignments were hidden from both participants and investigators until the end.

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

It started in 2016-02 with completion listed for 2018-03.

It ran at 2 sites in United States (2).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Incidence and Severity of Ocular TEAEs (time frame: Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16). The Incidence and Severity of Systemic Adverse Events (time frame: Screening Visit, Baseline (Day 1), Week 1, Week 4, Week 8, Week 12, and Week 16). Change From Baseline in Findings From Slit Lamp Examinations (SLE) Part A (time frame: Baseline, Week 1, Week 4, Week 8, Week 12, and Week 16).

It also tracked 18 secondary outcomes, for example Change From Baseline in Central Corneal Thickness by Visit Part A as Measured by Pachymetry for Part A, Central Corneal Thickness Part B, Change From Baseline Endothelial Cell Hexagonality in Percentage Over All 12 Weeks for Part A, Corneal Endothelial Cell Hexagonality Part B, Change From Baseline in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Scale for Part A and Best Corrected Visual Acuity (BCVA) Score Using ETDRS Scale for Part B.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2, randomized trial of SS-31 (Elamipretide) in 22 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.