Trial · Interventional
CompletedPhase 3Registry dataStudy 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
- Ph 3
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.
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)
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.
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.
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
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