Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) (NuPower)
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|ClinicalTrials.gov Identifier: NCT05162768|
Recruitment Status : Recruiting
First Posted : December 17, 2021
Last Update Posted : November 10, 2022
|Condition or disease||Intervention/treatment||Phase|
|Mitochondrial Myopathies Mitochondrial Pathology Mitochondrial DNA Mutation Mitochondrial Diseases Mitochondrial DNA Deletion Mitochondrial DNA Depletion Mitochondrial Metabolism Defect Mitochondrial Complex I Deficiency||Drug: Elamipretide Drug: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||130 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||
In this 48-week trial, subjects will be randomized (in a ratio of 1:1) to one of two groups: single daily subcutaneous doses of 60mg elamipretide, or, matching placebo, using a central randomization stratified by whether or not subjects have nPMD associated with replisome-related mutations.
130 subjects, consisting of 90 subjects with nPMD associated mutations of the mitochondrial replisome for primary analysis and an additional subset of up to 40 subjects who have nPMD associated with other non-replisome-related pathogenic mutations specific to nuclear DNA. Three periods: Screening (up to 28 days), Treatment (48 Weeks) and Follow-Up Period (4 weeks).
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||Trial personnel and subjects will be blinded to treatment until the database is locked. The Investigator will contact the Sponsor prior to unblinding any subject's treatment sequence unless in the instance of a medical emergency.|
|Official Title:||A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) NuPower|
|Actual Study Start Date :||April 29, 2022|
|Estimated Primary Completion Date :||April 2024|
|Estimated Study Completion Date :||June 2024|
0.75 mL of 80mg/mL solution of elamipretide for a single daily SC dose of 60mg elamipretide
60 mg of elamipretide administered as once daily 0.75 mL subcutaneous injections for 48 weeks
Other Name: MTP-131
Placebo Comparator: Placebo
0.75 mL of 80mg/mL solution of matching placebo for a single daily SC dose of 60mg
Placebo administered as once daily 0.75 mL subcutaneous injections for 48 weeks
- Six-minute walk test (6MWT) [ Time Frame: Baseline, Weeks 12, 24, 36, 48, 52 (End of Trial Visit) ]Change from Baseline in Distance Walked (in meters) on the Six-Minute Walk Test by Visit
- 5 times sit-to-stand test (5XSST) [ Time Frame: Baseline, Weeks 12, 24, 36, 48, 52 (End of Trial Visit) ]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) ]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 ]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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05162768
|Contact: Rekha Sathyanarayanaemail@example.com|