A Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of Bendavia™ (MTP-131) in Patients With Heart Failure

What Was Tested

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

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

The registry classes the interventions as drug.

Who 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: Diagnosis of New York Heart Association Class II or III HF for a minimum of 6 months prior to the Screening Visit. HF is considered to be stable, in the judgment of the Principal Investigator and no hospitalization related to HF has occurred within the 1 month prior to the Screening Visit. Females of child-bearing potential must have a negative serum pregnancy test at the Screening Visit. Written informed consent obtained that strictly adheres to the written guidelines from the local Ethical Committee (EC).

The full record lists 6 inclusion and 17 exclusion criteria; among the exclusions: coronary or peripheral artery revascularization procedure within 2 months prior to the screening visit; an acute myocardial infarction within 3 months prior to the screening visit; supine resting heart rate ≥ 100 beats per minute after 5 minutes rest.

How the Study Was Run

Design: randomized, parallel-group and triple-blind.

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 and investigator.

It started in 2015-05 with completion listed for 2015-09.

It ran at 1 site in Bulgaria.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Incidence of Adverse Events (time frame: Assessed up to Day 33). Mean peak plasma concentration (Cmax) of MTP-131 (ng/ml) in each cohort (time frame: Assessed up to Day 12).

It also tracked 1 secondary outcome, for example Changes in echocardiographic LV end-systolic volume (LVESV).

What This Means in Plain Language

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

It was withdrawn before enrolling anyone; no results are posted to the registry yet, so nothing here describes outcomes.

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