Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02805790
• Date of registration: 13/06/2016
• Prospective Registration: Yes
• Primary sponsor: Stealth BioTherapeutics Inc.
• Public title: Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study MMPOWER-2
• Scientific title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Study to Evaluate Safety, Tolerability, and Efficacy of Subcutaneous Injections of MTP-131 in Subjects With Mitochondrial Myopathy Previously Treated in the SPIMM-201 Study
• Date of first enrolment: August 22, 2016
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02805790
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Jim Carr
• Affiliation: Stealth BioTherapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Subject completed participation in the SPIMM-201 study without a significant protocol
deviation that would suggest the subject may not be able to complete all study
requirements in the opinion of the Sponsor

- Subject must reside in North America for the duration of the study

- Subject has not received study drug in the SPIMM-201 study within 3 weeks prior to
Screening

- Women of childbearing potential must agree to use 1 of the methods of birth control
specified in the protocol from the date they sign the informed consent form (ICF)
until two months after the last dose of study drug

- Subject has been on stable (unchanged and constant) medications (including over-the
counter treatments, vitamins, or supplements) for at least 1 month prior to the
Baseline Visit

Exclusion Criteria:

- 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 study requirements (i.e. unstable angina or recent myocardial infarction)

- Subject has received any investigational compound and/or has participated in another
interventional clinical study within 30 days prior to the Baseline Visit or is
concurrently enrolled in any non-interventional research of any type judged to be
scientifically or medically incompatible with the study as deemed by the Investigator
in consultation with the Sponsor

- Subject experienced an adverse reaction to study drug in the SPIMM-201 study that
contraindicates further treatment with elamipretide in the opinion of the Investigator
and/or Sponsor

- Female subjects who are pregnant, planning to become pregnant, or lactating

- Subject has undergone an in-patient hospitalization within the 1 month prior to the
Screening Visit or is likely to need in-patient hospitalization or a surgical
procedure during the course of the study

- Subject has a creatinine clearance =30 mL/min as calculated by the Cockcroft Gault
equation

- Subject has a corrected QT interval (QTc) elongation defined as a QTc >450 msec in
male subjects and >480msec in female subjects. Note: At the initial electrocardiogram
(ECG), if QTc exceeds these parameters, the ECG may be repeated 2 more times, and the
average of the 3 QTc values used to determine the subjects eligibility

- Subject has uncontrolled hypertension in the judgment of the Investigator (e.g.
elevated above >160 mmHg systolic or >100 mmHg diastolic despite appropriate treatment
on two consecutive readings)

- Subject has a history of clinically significant hypersensitivity or allergy to any of
the excipients contained in the study drug

- Subject has a history of active alcoholism or drug addiction during the year before
the Screening Visit



Age minimum: 16 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primary Mitochondrial Disease

Intervention(s)

Drug: Elamipretide

Drug: Placebo

Primary Outcome(s)

Distance Walked on the 6-minute Walk Test (6MWT) by Visit [Time Frame: End of Week 4 and End of Week 12]

Secondary Outcome(s)

Neuro-QoL Fatigue Short Form Score: Total T-Scores (Question 1-8) [Time Frame: End of Week 4 and End of Week 12]

Patient Global Assessment by Visit, Categorical [Time Frame: End of Week 4 and End of Week 12]

Physician Global Assessment (PhGA) By Visit, Categorical [Time Frame: End of Week 4 and End of Week 12]

Wrist Accelerometer Counts by Day [Time Frame: Last 7 days prior to the date of end of treatment visit]

Physician Global Assessment (PhGA) By Visit, Continuous [Time Frame: End of Week 4 and End of Week 12]

Average Hip Accelerator Counts by Day [Time Frame: Last 7 days prior to the date of end of treatment visit]

Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) Total Fatigue Score by Week [Time Frame: Last 7 days of Week 4 and Last 7 days of Week 12]

Patient Global Assessment [PGA] Score by Visit, Continuous [Time Frame: End of Week 4 and End of Week 12]

Triple Timed Up and Go (3TUG) Test by Visit [Time Frame: End of Week 4 and End of Week 12]

Secondary ID(s)

SPIMM-202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 07/07/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02805790