A Study to Evaluate the Effects of 4 Weeks Treatment With Subcutaneous Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Preserved Ejection Fraction

What Was Tested

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

This is a multi-center, randomized, double-blind, placebo-controlled study in subjects with stable heart failure with preserved ejection fraction (HFpEF) to evaluate the effects of 4 weeks treatment with subcutaneous MTP-131 (elampretide) on left ventricular function.

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

Placebo once daily for 28 consecutive days.

The registry classes the interventions as drug.

Who Took Part

46 actual participants took part.

It was open to people of any sex aged from 40 years up to 80 years.

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

Examples of who could join: Age ≥45 and \<80 years. Symptomatic heart failure (i.e. NYHA II or III) due to HFpEF for at least 6 months prior to study start. Women of child-bearing potential must have a negative serum pregnancy test at baseline. Willing and able to provide signed informed consent form (ICF) prior to participation in any study-related procedures.

The full record lists 9 inclusion and 14 exclusion criteria; among the exclusions: probable alternative diagnoses that in the opinion of the investigator could account for the patient's symptoms e.g. severe pulmonary dysfunction or severe asthma; coronary or peripheral revascularization procedures, valvular procedures, or any major surgical procedure within 3 months prior to the screening visit; acute coronary syndrome (acs), stroke or transient ischemic attack (tia) within 3 months prior to the screening visit.

How the Study Was Run

The set-up was randomized, parallel-group and quadruple-blind.

The participants were allocated by chance, so the SS-31 (Elamipretide) group and the comparison group should start out alike.

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

Neither the participants nor the staff measuring them knew who was on SS-31 (Elamipretide), so expectations could not easily colour the readings.

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

It started in 2016-09-02 with completion listed for 2017-05-04.

It ran at 9 sites in Serbia (7) and Germany (2).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Compare the delta in E/e' at rest exercise measured with echocardiography between the elamipretide and placebo groups (time frame: 4 weeks).

It also tracked 4 secondary outcomes, for example Compare the change in 6-minute walking distance, between the elamipretide and placebo groups at the end of the 4-week treatment period and Compare the change in NT-proBNP, between the elamipretide and placebo groups at the end of the 4-week treatment period.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of SS-31 (Elamipretide) in 46 people.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

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