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Trial record 1 of 2 for:    Mmpower 2
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A Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Mitochondrial Disease Previously Treated in the Stealth BioTherapeutics SPIMM-201 Study (MMPOWER-2)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02805790
First Posted: June 20, 2016
Last Update Posted: January 24, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.
  Purpose
This is a Phase 2, randomized, double-blind, placebo-controlled crossover study which will enroll up to 36 subjects (anticipated) with genetically confirmed mitochondrial disease who have completed participation in the SPIMM-201 (MMPOWER) study. Subjects will be randomized (1:1) to one of two sequence groups: 4-weeks of treatment with 40 mg elamipretide administered once daily subcutaneously in Treatment Period 1 followed by 4-weeks of treatment with placebo administered once daily subcutaneously in Treatment Period 2 (separated by 4-week washout period), or vice versa

Condition Intervention Phase
Primary Mitochondrial Disease Drug: Elamipretide Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Mitochondrial Disease Previously Treated in the Stealth BioTherapeutics SPIMM-201 Study

Resource links provided by NLM:


Further study details as provided by Stealth BioTherapeutics Inc.:

Primary Outcome Measures:
  • Distance walked (meters) on the 6-minute walk test (6MWT) [ Time Frame: 4 weeks ]

Secondary Outcome Measures:
  • Incidence of adverse events [ Time Frame: 14 weeks ]
  • Change in vital signs [ Time Frame: baseline through 14 weeks ]
  • Changes in electrocardiograms ECGs [ Time Frame: 14 weeks ]
  • Changes in clinical laboratory evaluations [ Time Frame: 14 weeks ]
  • Changes in accelerometer counts (wrist) [ Time Frame: 10 weeks ]
  • Changes in accelerometer counts (hip) [ Time Frame: 10 weeks ]
  • Changes in the Triple Timed Up and Go score [ Time Frame: 4 weeks ]
  • Changes in exploratory biomarkers [ Time Frame: 14 weeks ]
  • Changes in the NeuroQOL Fatigue Questionnaire [ Time Frame: 14 weeks ]
  • Changes in the Patient Global Assessment [ Time Frame: 14 weeks ]
  • Changes in the Physician Global Assessment [ Time Frame: 14 weeks ]

Enrollment: 30
Study Start Date: July 2016
Estimated Study Completion Date: May 2017
Estimated Primary Completion Date: March 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Drug
Patients will be randomly assigned to receive 40 mg of elamipretide in either Treatment Period 1 or Treatment Period 2.
Drug: Elamipretide
40 mg elamipretide administered once daily subcutaneously
Other Names:
  • Bendavia
  • MTP-131
Placebo Comparator: Placebo
Patients will be randomly assigned to receive placebo comparator either in Treatment Period 1 or Treatment Period 2
Drug: Placebo
4 weeks of treatment with placebo administered once daily subcutaneously
Other Names:
  • Bendavia
  • MTP-131

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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 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
  Contacts and Locations
Information from the National Library of Medicine

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): NCT02805790


Locations
United States, California
University of California
San Diego, California, United States
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States
United States, Ohio
Akron Children's Hospital
Akron, Ohio, United States
United States, Pennsylvania
Children's Hospital of Pittsburg of UPMC
Pittsburgh, Pennsylvania, United States
Sponsors and Collaborators
Stealth BioTherapeutics Inc.
  More Information

Responsible Party: Stealth BioTherapeutics Inc.
ClinicalTrials.gov Identifier: NCT02805790     History of Changes
Other Study ID Numbers: SPIMM-202
First Submitted: June 13, 2016
First Posted: June 20, 2016
Last Update Posted: January 24, 2017
Last Verified: January 2017

Keywords provided by Stealth BioTherapeutics Inc.:
Primary Mitochondrial Disease
Mitochondrial Myopathy
Elamipretide
MTP-131
Bendavia™

Additional relevant MeSH terms:
Mitochondrial Diseases
Metabolic Diseases