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Trial record 1 of 3 for:    MMPOWER
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A Study Investigating the Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy (MMPOWER)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02367014
First Posted: February 20, 2015
Last Update Posted: June 1, 2016
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 study will be a Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study, enrolling an anticipated 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of MTP-131 in this patient population.

Condition Intervention Phase
Mitochondrial Myopathy Drug: MTP-131 Drug: Placebo Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study Investigating the Safety, Tolerability, and Efficacy of Intravenous MTP-131 for the Treatment of Mitochondrial Myopathy in Subjects With Genetically Confirmed Mitochondrial Disease

Resource links provided by NLM:


Further study details as provided by Stealth BioTherapeutics Inc.:

Primary Outcome Measures:
  • Incidence of Adverse Events [ Time Frame: Assessed at each visit from Baseline to Day 7 ]
  • Changes in vital signs [ Time Frame: Assessed at each visit from Baseline to Day 7, except on Day 6 ]
  • Changes in clinical laboratory evaluations [ Time Frame: Assessed at each visit from Baseline to Day 7, except on Day 6 ]
  • Change in distance walked (meters) on the 6-minute walk test (6MWT) [ Time Frame: Assessed at Baseline, Day 5 (end-of-treatment visit) and Day 7 ]

Secondary Outcome Measures:
  • Change in selected outputs of Cardiopulmonary Exercise Testing (CPET) [ Time Frame: Assessed at Baseline, Day 5 (end-of-treatment visit) and Day 7 ]

Enrollment: 36
Study Start Date: February 2015
Study Completion Date: May 2016
Primary Completion Date: April 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Low Dose Drug: MTP-131
MTP-131 (low dose) administered as single day intravenous infusion over 2 hours for 5 days
Experimental: Intermediate dose Drug: MTP-131
MTP-131 (intermediate dose) administered as single day intravenous infusion over 2 hours for 5 days
Experimental: High dose Drug: MTP-131
MTP-131 (high dose) administered as single day intravenous infusion over 2 hours for 5 days
Placebo Comparator: Placebo Drug: Placebo
Placebo Comparator (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of mitochondrial disease believed to impair the mitochondrial respiratory chain.
  • Eligibility requires prior genetic confirmation of mitochondrial disease.
  • Diagnosis of mitochondrial myopathy judged by the Investigators to be due to existing mitochondrial disease.
  • Subject must be able to complete a Screening Visit 6MWT.
  • Women of childbearing potential must agree to use birth control as specified in the protocol from the date they sign the ICF until two months after the last dose of study drug.
  • Eligible subjects may be required to stay overnight in the clinical research center for the duration of their study participation lasting up to 7 consecutive days.

Exclusion Criteria:

  • 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.
  • Hospitalized (admitted as in-patient) within 1 month prior to the Baseline Visit.
  • A history of type 1 diabetes mellitus (T1DM).
  • Uncontrolled Type 1 (T1DM) or Type 2 diabetes mellitus (T2DM), in the opinion of the investigator.
  • A creatinine clearance <45 mL/min as calculated by the Cockcroft Gault equation.
  • Requires pacemaker, defibrillator, or has undergone cardiac surgery within 2 years of the Screening Visit.
  • Uncontrolled hypertension (>160 mmHg systolic or >100 mmHg diastolic) at the Screening Visit.
  • Subject has participated in another interventional clinical trial within 3 months of the screening visit or is currently enrolled in a non-interventional clinical trial judged by the Investigator to be incompatible with the current trial.

    • Other protocol-defined inclusion/exclusion criteria may apply.
  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): NCT02367014


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: NCT02367014     History of Changes
Other Study ID Numbers: SPIMM-201
First Submitted: February 9, 2015
First Posted: February 20, 2015
Last Update Posted: June 1, 2016
Last Verified: May 2016

Keywords provided by Stealth BioTherapeutics Inc.:
Mitochondrial Myopathy
Primary Mitochondrial Disease
Bendavia™
elamipretide

Additional relevant MeSH terms:
Muscular Diseases
Mitochondrial Diseases
Mitochondrial Myopathies
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Metabolic Diseases