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Trial · Interventional

CompletedPhase 3Registry data

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

Short name NuPower

Overview

Status
Completed
Phase
Phase 3
Enrollment
102 participants (actual)
Sponsor
Stealth BioTherapeutics Inc. · Industry
Design
Randomized · Quadruple masking
Countries
10 countries

United States, Australia, Germany, Hungary, Italy, Netherlands, New Zealand, Norway, Spain, United Kingdom

Start
29 Apr 2022
Primary completion
30 Sept 2024
Completion
4 Dec 2024
First posted
17 Dec 2021
Last update posted
24 Oct 2025
Conditions
Mitochondrial Myopathies · Mitochondrial Pathology · Mitochondrial DNA Mutation · Mitochondrial Diseases · Mitochondrial DNA Deletion · Mitochondrial DNA Depletion · Mitochondrial Metabolism Defect · Mitochondrial Complex I Deficiency

Timeline

1 trial · 1 completed

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2022202320242025
  1. Ph 3

    NuPower

    NCT05162768

    29 Apr 2022 to 4 Dec 2024

Registry summary

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

SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide as a treatment for subjects with primary mitochondrial myopathy associated with nuclear DNA mutations (nPMD).

Interventions

  • ElamipretideDrug
  • PlaceboDrug

Arms2

  • Experimental

    Elamipretide

    0.75 mL of 80mg/mL solution of elamipretide for a single daily SC dose of 60mg elamipretide

  • Placebo comparator

    Placebo

    0.75 mL of 80mg/mL solution of matching placebo for a single daily SC dose of 60mg

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Six-minute walk test (6MWT)

    Time frame · Baseline, Weeks 12, 24, 36, 48, 52 (End of Trial Visit)

    Registry description

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

Secondary outcomes(3)
  1. 5 times sit-to-stand test (5XSST)

    Time frame · Baseline, Weeks 12, 24, 36, 48, 52 (End of Trial Visit)

    Registry description

    Change from Baseline in Total time (in seconds) to complete the 5XSST. Participant is directed to stand up straight as quickly as possible 5 times, without stopping in between, keeping arms folded across the chest. An average time is calculated.

  2. Triple Timed up-and-go test (3TUG)

    Time frame · Baseline, Weeks 12, 24, 36, 48, 52 (End of Trial Visit)

    Registry description

    Change from Baseline in Total time (in seconds) to complete the 3TUG. Participant is directed to stand up from chair, walk at normal pace to the line on the floor 3 meters away, turn, walk back to the chair at normal pace, sit down again; activity is timed, in seconds. Activity is repeated 3 times consecutively without rest and an average time is calculated.

  3. Patient Global Impression of Severity (PGI-S) Scale

    Time frame · Baseline, Weeks 12, 24, 36, 48

    Registry description

    Change from Baseline for PGI of Severity (PGI-S) Scale. Patient-reported current health status by week and at end of treatment. PGI-S Scale is a categorical scale and asks the participant to "rate the severity of your muscle weakness symptoms today" as one of the following categories: None, Mild, Moderate, Severe, or Very Severe. None means better health status, and best outcome, Very severe means worse health status and worse outcome.

Posted results

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

No results are posted for this trial.

Completed trials do not always post results.

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

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 18 Years up to 70 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: A subject must meet all of the following inclusion criteria at the Screening and Baseline Visit (unless otherwise specified) to be eligible for inclusion in the SPIMD-301 trial: 1. Willing and able to provide a signed informed consent form (ICF) prior to participation in any trial-related procedures. 2. Agrees and is able to adhere to the trial requirements for the length of the trial, including administration of assigned treatment. 3. Is ≥18 years and ≤ 70 years of age at the time of screening. 4. Diagnosed with nPMD with a predominant clinical manifestation of myopathy, which must include progressive external ophthalmoplegia (PEO) and exercise intolerance and/or skeletal muscle weakness, with genetic confirmation of either: 1. Nuclear DNA mutation of the mitochondrial replisome (replisome-related mutations), which include the following genes: * POLG 1/2 * TWINKLE (C10ORF2) * TYMP * DGUOK * TK2 * RRM2B * RNASEH1 * SSBP * MGME1 * DNA2 * ANT1 (SLC25A4) * SUCLG1 * SUCLA2 * MPV17 or 2. Other pathogenic mutations specific to nuclear DNA. 5. Women of childbearing potential must agree to use one of the following methods of birth control from the date they sign the ICF until 28 days after the last dose of IMP: 1. Abstinence, when it is in line with the preferred and usual lifestyle of the subject. Subject agrees to use a highly effective method of contraception should they become sexually active. 2. Relationships with male partners who have been surgically sterilized by vasectomy (the vasectomy procedure must have been conducted at least 60 days prior to the Screening Visit). 3. Barrier method (e.g., condom or occlusive cap) with spermicidal foam/gel/film/cream AND either hormonal contraception (oral, implanted, or injectable) or an intrauterine device or system. Note: Non-childbearing potential is defined as surgical sterilization (e.g., bilateral oophorectomy, hysterectomy, or tubal ligation) or postmenopausal (defined as permanent cessation of menstruation for at least 12 consecutive months prior to the Screening Visit). 6. Male subjects with female partners of childbearing potential must be willing to use a highly effective method of contraception from the date they sign the ICF until 28 days after the last dose of IMP. Exclusion Criteria: 1. Is unable to perform the 6MWT, 3TUG, or 5XSST functional tests. The use of a gait assist device is allowed; however, use should remain consistent for the entire duration of the trial. 2. Female subjects who are pregnant, planning to become pregnant, or breastfeeding/lactating. 3. Walks \< 150 meters or \> 450 meters during the 6MWT (Screening Visit only). 4. The estimated glomerular filtration rate (eGFR) is \< 30 mL/min/1.73 m2, using the Modification of Diet in Renal Disease (MDRD) Study equation (Screening Visit only). 5. Has undergone an in-patient hospitalization within 30 days prior to screening or has a planned hospitalization or a surgical procedure during the trial, unless, in the opinion of the Investigator, it is concluded that it will not impact the outcome measurements of the trial. 6. Has clinically significant respiratory disease and/or cardiac disease that would interfere with trial assessments, in the opinion of the Investigator. 7. Has had any prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to screening. 8. Has history of or current severe neurologic impairment, severe epilepsy, severe ataxia, or severe neuropathy that may interfere with their ability to complete all trial requirements, in the opinion of the Investigator. 9. Active malignancy or any other cancer from which the subject has been disease-free for \< 2 years. Localized squamous or non-invasive basal cell skin carcinomas are allowed, if appropriately treated prior to screening. 10. Has had a solid organ transplant. 11. Has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection. 12. Has a history of a systemic eosinophilic illness and/or an eosinophil count \>1,000 cells x106/L at the Screening Visit. 13. 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 prior to current trial; or is currently enrolled in a non-interventional clinical trial that, in the opinion of the Investigator, may be potentially confounding to the results of the current trial (e.g., exercise therapy trial). 14. Has received elamipretide (MTP-131) within the past one year of the Screening Visit. 15. Has a history of active substance abuse during the year prior, in the opinion of the Investigator.

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