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A Trial to Evaluate the Safety and Efficacy of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Extension (MMPOWER-3)

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ClinicalTrials.gov Identifier: NCT03323749
Recruitment Status : Active, not recruiting
First Posted : October 27, 2017
Last Update Posted : July 18, 2019
Sponsor:
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.

Tracking Information
First Submitted Date  ICMJE October 12, 2017
First Posted Date  ICMJE October 27, 2017
Last Update Posted Date July 18, 2019
Actual Study Start Date  ICMJE October 27, 2017
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 24, 2017)
  • Change in distance walked on the six minute walk test (6MWT) [ Time Frame: Baseline to 24 weeks ]
  • Change in total fatigue score on the on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) [ Time Frame: Baseline to 24 weeks ]
    Each individual item score ranges from 1 (none) to 4 (severe). The total fatigue score ranges from 4-16. Lower values represent a better outcome. The total fatigue score is the sum of question 1 through question 4 on the Primary Mitochondrial Myopathy Symptom Assessment.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03323749 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 24, 2017)
  • Change in fatigue during activities score on the Primary Mitochondrial Disease Symptom Assessment (PMMSA) [ Time Frame: Baseline to 24 weeks ]
    Each individual item score ranges from 1 (none) to 4 (severe). Lower values represent a better outcome. The total fatigue during activities score is the sum of question 2 and question 4 on the Primary Mitochondrial Myopathy Symptom Assessment.
  • Change in the Neuro-QoL Fatigue Item Bank score [ Time Frame: Baseline to 24 weeks ]
    Each individual item score ranges from 1-5. Total raw score for the entire item bank ranges from 19-95. Raw scores will be calibrated using Item Response Theory Model. Lower values represent a better outcome. Individual items will be summed to calculate total scores.
  • Change in the most bothersome symptom score on the Primary Mitochondrial Myopathy Symptoms Assessment [ Time Frame: Baseline to 24 weeks ]
    The item score rangers from 1 (none) to 4 (severe). Lower values represent a better outcome. The most bothersome score is the average of the identified most bothersome symptom of the Primary Mitochondrial Myopathy Symptom Assessment by each subject.
  • Evaluate safety and tolerability through incidence of treatment emergent adverse events [ Time Frame: Baseline to 28 weeks ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: October 24, 2017)
Part 2: Evaluate the long term safety and tolerability of elamipretide using the elamipretide delivery system through incidence of treatment emergent adverse events [ Time Frame: Starting at week 24 up to 144 weeks ]
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE A Trial to Evaluate the Safety and Efficacy of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Extension
Official Title  ICMJE 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 Myopathy Followed by an Open-Label Treatment Extension
Brief Summary This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial myopathy. This will be followed by an open-label treatment extension.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Primary Mitochondrial Myopathy
Intervention  ICMJE
  • Combination Product: elamipretide
    40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
    Other Name: MTP-131
  • Combination Product: placebo comparator
    40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
    Other Name: Placebo
  • Combination Product: elamipretide open label treatment
    40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system
Study Arms  ICMJE
  • Experimental: Part 1: Elamipretide
    Subjects will be randomized to receive either elamipretide or placebo for 24 weeks. For the elamipretide arm, subjects will administer daily 40 mg (0.5mL) subcutaneous injections of elamipretide.
    Intervention: Combination Product: elamipretide
  • Placebo Comparator: Part 1: Placebo
    Subjects will be randomized to receive either elamipretide or placebo for 24 weeks. For the placebo arm, subjects will administer daily 40 mg (0.5 mL) subcutaneous injections of placebo for 24 weeks.
    Intervention: Combination Product: placebo comparator
  • Experimental: Part 2: Elamipretide open label
    Once subjects complete part 1, and meet continuation criteria, they will have the option to continue into an open-label treatment extension. Subjects will receive 40 mg (0.5 mL) subcutaneous injections of elamipretide for up to 144 weeks.
    Intervention: Combination Product: elamipretide open label treatment
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: July 17, 2019)
218
Original Estimated Enrollment  ICMJE
 (submitted: October 24, 2017)
202
Estimated Study Completion Date  ICMJE August 2022
Estimated Primary Completion Date December 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

PART 1:

Inclusion Criteria:

  • Willing and able to provide a signed informed consent form prior to participation in any trial-related procedures
  • Agrees to adhere to the trial requirements for the length of the trial, including the use of the elamipretide delivery system
  • Subject is ≥ 16 and ≤ 80 years of age
  • Diagnosed with PMM in the opinion of the investigator and confirmed by an Adjudication Committee
  • Woman of childbearing potential must agree to use a highly effective method of birth control

Exclusion Criteria:

  • Subject has myopathic signs and or/symptoms due to a neuropathic process or gait problem that would interfere with the 6MWT, in the opinion of the Investigator
  • Female who are pregnant, planning to become pregnant, or breastfeeding/lactating
  • At Screening, the estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m^2
  • Subject has undergone an in-patient hospitalization within the 30 days prior to the Baseline Visit or has a planned hospitalization or a surgical procedure during the trial.
  • Subject has clinically significant cardiac disease or prior interventional procedure and/or respiratory disease (medical history or current clinical findings) within 3 months of the Baseline Visit, in the opinion of the Investigator.
  • Subject has QTc elongation (using the correction factor utilized at the clinical site) defined as a QTc >450 msec in male subjects and >480 msec in female subjects.
  • ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following:

    1. First degree AV-block
    2. Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
    3. Right bundle branch block
  • Subject has severe vision impairment that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements
  • Subject has a seizure disorder that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements.
  • Active malignancy or any other cancer from which the subject has been disease-free for < 2 years.
  • Subject has a solid organ transplant and/or is currently receiving treatment with therapy for immunosuppression, in the opinion of the Investigator.
  • Subject has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection.
  • Subject has a history of a systemic eosinophilic illness and/or an eosinophil count >1,000 cells x10^6/L at the Screening Visit.
  • Subject 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 of the Baseline Visit; or is currently enrolled in a non-interventional clinical trial (except for SPIMM-300) at the Baseline Visit which, in the opinion of the Investigator, may be potentially confounding with results of the current trial (e.g., exercise therapy trial).
  • Subject has previously received elamipretide (MTP-131), for any reason.
  • Subject has a history of active substance abuse during the year before the Baseline Visit, in the opinion of the Investigator.
  • 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 trial requirements.

PART 2:

Continuation Criteria:

  • Subjects must continue to be able and willing to adhere to the trial requirements.
  • Subject is appropriate to continue in Part 2 (i.e. subject was compliant in Part 1), in the opinion of the Investigator.
  • Subject has not had a serious adverse event (SAE)/serious adverse device effect (SADE) attributed to the elamipretide delivery system.
  • Subject has not permanently discontinued the elamipretide delivery system.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 16 Years to 80 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Denmark,   Germany,   Hungary,   Italy,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03323749
Other Study ID Numbers  ICMJE SPIMM-301
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Stealth BioTherapeutics Inc.
Study Sponsor  ICMJE Stealth BioTherapeutics Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Stealth BioTherapeutics Inc.
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP