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Trial · Interventional

TerminatedPhase 3Results postedRegistry data

A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension

Short name MMPOWER-3

Overview

Status
Terminated
Phase
Phase 3
Enrollment
218 participants (actual)
Sponsor
Stealth BioTherapeutics Inc. · Industry
Design
Randomized · Quadruple masking
Countries
7 countries

United States, Canada, Denmark, Germany, Hungary, Italy, United Kingdom

Start
9 Oct 2017
Primary completion
10 Feb 2020
Completion
10 Feb 2020
First posted
27 Oct 2017
Last update posted
24 Jan 2022
Conditions
Primary Mitochondrial Myopathy

Timeline

1 trial · 1 stopped early · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20172018201920202021
  1. Ph 3

    MMPOWER-3

    NCT03323749

    9 Oct 2017 to 10 Feb 2020

    Terminated

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial myopathy. This will be followed by an open-label treatment extension.

Interventions

  • elamipretideCombination product
  • placebo comparatorCombination product
  • elamipretide open label treatmentCombination product

Arms3

  • Experimental

    Part 1: Elamipretide

    40 mg (0.5mL) elamipretide subcutaneous (SC) daily

  • Placebo comparator

    Part 1: Placebo

    Placebo SC daily

  • Experimental

    Part 2: Elamipretide open label

    Elamepretide 40 mg (0.5 mL) SC daily

Primary outcomes2

Outcomes the sponsor listed as primary.

  1. Six-minute Walk Test (6MWT)

    Time frame · Baseline to 24 weeks

    Registry description

    Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit

  2. Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)

    Time frame · Baseline to 24 weeks

    Registry description

    Change from Baseline in Total fatigue score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) by visit. Each individual item score ranges from 1 (none) to 4 (severe). The total fatigue score ranges from 4-16. Lower values represent a better outcome. The total fatigue score is the sum of question 1 through question 4 on the Primary Mitochondrial Myopathy Symptom Assessment.

Secondary outcomes(4)
  1. Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA).

    Time frame · Baseline to 24 weeks

    Registry description

    Change from baseline in Fatigue During Activities. Fatigue During Activities is the sum of question 2 (tiredness during activities) and question 4 (muscle weakness during activities.) The four response options are: 1=Not at all, 2=Mild, 3=Moderate, and 4=Severe. Raw scores for each subject range from 2-8. A lower score means a better outcome, with less fatigue. A higher score means a worse outcome, with more fatigue.

  2. Neuro-QoL Fatigue Activities of Daily Living

    Time frame · Baseline to 24 weeks

    Registry description

    Change From Baseline in Neuro-QoL Fatigue Activities of Daily Living by Visit. Each individual item score ranges from 1-5. Total raw score for the entire item bank ranges from 19-95. Raw scores will be calibrated using Item Response Theory Model. Lower values represent a better outcome. Individual items will be summed to calculate total scores.

  3. Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment

    Time frame · Baseline to 24 weeks

    Registry description

    The item score rangers from 1 (none) to 4 (severe). Lower values represent a better outcome. The most bothersome score is the average of the identified most bothersome symptom of the Primary Mitochondrial Myopathy Symptom Assessment by each subject.

  4. Neuro-QoL Fatigue Short Form Score

    Time frame · 24 Weeks

    Registry description

    Change From Baseline in Neuro-QoL Fatigue - Short Form: Total T-Scores by Visit. The Neuro-QoL Fatigue Short Form is comprised of the sum of the first 8 questions of the Neuro-QoL Item Bank v1.0 - Fatigue. Each question is scored as following: 1=Never, 2=Rarely, 3=Sometimes, 4=Often, and 5=Always. The questions include: I felt exhausted, I felt that I had no energy, I felt fatigued, I was too tired to do my household chores, I was too tired to leave the house, I was frustrated by being too tired to do the things I wanted to do, I felt tired, and I had to limit my social activity because I was tired. T-scores are calculated from the short form scoring table provided by the instrument authors (Neuro-QoL User Manual, 2015). T-score distributions rescale raw scores into standardized scores with a mean of 50 and a standard deviation (SD) of 10. Change from baseline: Negative numbers mean less fatigue, better outcome, positive score means more fatigue, worse outcome.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· Baseline to 24 weeks

Six-minute Walk Test (6MWT)

GroupValue (meters)Spread
Elamipretide16.24342.0071
Placebo7.81139.5737
Primary outcomeMean· Baseline to 24 weeks

Total Fatigue Score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)

GroupValue (score on a scale)Spread
Elamipretide-1.011.714
Placebo-1.081.865
Secondary and other outcome results(4)
Secondary outcomeMean· Baseline to 24 weeks

Fatigue During Activities Score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA).

GroupValue (score on a scale)Spread
Elamipretide-0.480.890
Placebo-0.600.998
Secondary outcomeMean· Baseline to 24 weeks

Neuro-QoL Fatigue Activities of Daily Living

GroupValue (score on a scale)Spread
Elamipretide-2.55.33
Placebo-2.75.31
Secondary outcomeMean· Baseline to 24 weeks

Change From Baseline in the Most Bothersome Symptom Score on the Primary Mitochondrial Myopathy Symptoms Assessment

GroupValue (score on a scale)Spread
Elamipretide-0.210.492
Placebo-0.240.535
Secondary outcomeMean· 24 Weeks

Neuro-QoL Fatigue Short Form Score

GroupValue (score on a scale)Spread
Elamipretide-3.375.077
Placebo-2.895.915

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Double-Blind ElamipretideDouble-blind period: elamipretide: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then elamipretide open-label treatment: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system0 / 1095 / 109107 / 109
Double-Blind PlaceboPlacebo SC daily Double-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system.0 / 1093 / 10983 / 109
Open-Label ElamipretideDouble blind period: elamipretide: 40mg (0.5mL) of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system, then elamipretide open-label treatment: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system0 / 9312 / 9388 / 93
Open-Label PlaceboDouble-blind period: placebo comparator: Placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system. Open-label period: 40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system.0 / 1039 / 103102 / 103

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies2

Studies that cite this trial.

Studies with results

2 studies on this page

Indexing hint: Randomized controlled trial · 2024

3 results

Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial.

Karaa et al. 2024Orphanet journal of rare diseasescited by 11

PMID 39574155PMC11583740DOI 10.1186/s13023-024-03421-5PUB-39574155

CC BY3 results

Indexing hint: Randomized controlled trial · 2023

3 results

Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial.

Karaa et al. 2023Neurologycited by 66

PMID 37268435PMC10382259DOI 10.1212/wnl.0000000000207402PUB-37268435

CC BY3 results

Eligibility

Who could join, as posted.

Sex
All
Age
from 16 Years up to 80 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
PART 1: Inclusion Criteria: * Willing and able to provide a signed informed consent form prior to participation in any trial-related procedures * Agrees to adhere to the trial requirements for the length of the trial, including the use of the elamipretide delivery system * Subject is ≥ 16 and ≤ 80 years of age * Diagnosed with PMM in the opinion of the investigator and confirmed by an Adjudication Committee * Woman of childbearing potential must agree to use a highly effective method of birth control Exclusion Criteria: * Subject has myopathic signs and or/symptoms due to a neuropathic process or gait problem that would interfere with the 6 minute walk test (6MWT), in the opinion of the Investigator * Female who are pregnant, planning to become pregnant, or breastfeeding/lactating * At Screening, the estimated glomerular filtration rate (eGFR) \< 30 mL/min/1.73 m\^2 * Subject has undergone an in-patient hospitalization within the 30 days prior to the Baseline Visit or has a planned hospitalization or a surgical procedure during the trial. * Subject has clinically significant cardiac disease or prior interventional procedure and/or respiratory disease (medical history or current clinical findings) within 3 months of the Baseline Visit, in the opinion of the Investigator. * Subject has QTc elongation (using the correction factor utilized at the clinical site) defined as a QTc \>450 msec in male subjects and \>480 msec in female subjects. * ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following: 1. First degree Atrioventricular bock (AV-block) 2. Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type) 3. Right bundle branch block * Subject has severe vision impairment that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements * Subject has a seizure disorder that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements. * Active malignancy or any other cancer from which the subject has been disease-free for \< 2 years. * Subject has a solid organ transplant and/or is currently receiving treatment with therapy for immunosuppression, in the opinion of the Investigator. * Subject has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection. * Subject has a history of a systemic eosinophilic illness and/or an eosinophil count \>1,000 cells x10\^6/L at the Screening Visit. * Subject is currently participating or has participated in an interventional clinical trial (i.e.,investigational product or device, stem cell therapy, gene therapy) within 30 days of the Baseline Visit; or is currently enrolled in a non-interventional clinical trial (except for SPIMM-300) at the Baseline Visit which, in the opinion of the Investigator, may be potentially confounding with results of the current trial (e.g., exercise therapy trial). * Subject has previously received elamipretide (MTP-131), for any reason. * Subject has a history of active substance abuse during the year before the Baseline Visit, in the opinion of the Investigator. * Subject has any prior or current medical condition that, in the judgment of the Investigator, would prevent the subject from safely participating in and/or completing all trial requirements. PART 2: Continuation Criteria: * Subjects must continue to be able and willing to adhere to the trial requirements. * Subject is appropriate to continue in Part 2 (i.e. subject was compliant in Part 1), in the opinion of the Investigator. * Subject has not had a serious adverse event (SAE)/serious adverse device effect (SADE) attributed to the elamipretide delivery system. * Subject has not permanently discontinued the elamipretide delivery system.

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