An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration

What Was Tested

The trial (phase 1) is built around SS-31 (Elamipretide).

This is an open-label, Phase 1 single-center study in approximately 40 subjects who have 1 eye with intermediate AMD, including a high-risk drusen without geographic atrophy (GA) subgroup and a noncentral GA subgroup.

It had two arms (groups): Intermediate AMD - HRD without GA (experimental) and Intermediate AMD with NCGA (experimental).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 40 actual participants took part.

It was open to people of any sex aged from 55 years with no upper age limit.

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

Examples of who could join: Adults ≥ 55 years of age with 1 eye with intermediate AMD - noncentral GA. No evidence of choroidal neovascularization (active or prior history) in the study eye. Geographic atrophy may be multifocal, but the cumulative GA lesion size must be:. ≥ 1.27 mm2 (approximately ≥ 0.5 DA) and ≤ 10.16 mm2 (approximately ≤ 4 DA). Must reside completely within the FAF imaging field (field 2 to 30-degree image centered on the fovea).

The full record lists 21 inclusion and 30 exclusion criteria; among the exclusions: age-related macular degeneration with any evidence of central ga (i.e., involving the fovea); atrophic retinal disease because of causes other than amd; presence or diagnosis of exudative amd or choroidal neovascularization in the study eye.

How the Study Was Run

Design: non-randomized, single-group and open-label.

Everyone knew when SS-31 (Elamipretide) was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2016-10-28 with completion listed for 2018-03-16.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Change From Baseline in Mean Standard Luminance Best-corrected Visual Acuity (BCVA) (time frame: Day 0 (Baseline to Day 7, and to Weeks 4, 8, 12, 16, 20, 24, and 28).

It also tracked 13 secondary outcomes, for example Change From Baseline in Mean Low Luminance Best-Corrected Visual Acuity (LLBCVA), Change From Baseline in Mean Dark Adaptometry, Mean Treatment Compliance (%) of Administration of Subcutaneous Elamipretide, Mean Number of Home Health Visits to Administer Elamipretide, Change From Baseline in Mean Area of Geographic Atrophy by Fundus Autofluorescence and Change From Baseline in Mean Area of Geographic Atrophy as Measured by Spectral-Domain Optical Coherence Tomography (SD-OCT).

What This Means in Plain Language

Put simply, this is a phase 1, non-randomized trial of SS-31 (Elamipretide) in 40 people.

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.