Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study
Condition: Primary Mitochondrial Disease
MeSH terms: Mitochondrial Myopathies; Mitochondrial Diseases
Intervention: Elamipretide; Placebo
Intervention description: 4 weeks of treatment with 40 mg elamipretide administered once daily subcutaneously; 4 weeks of treatment with placebo administered once daily subcutaneously
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 30.0
Sex: All
Minimum age: 16 Years
Healthy volunteers: No
Keywords: Primary Mitochondrial Disease; Mitochondrial Myopathy; Elamipretide; MTP-131; Bendavia™

Summary:

      This randomized, double-blind, placebo-controlled, crossover study screened 32 subjects with
      primary mitochondrial myopathy (PMM) to evaluate the safety, tolerability, pharmacokinetics
      (PK), and efficacy of subcutaneous elamipretide in this patient population.
    

Eligibility 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