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

NCT02805790 | Completed Phase 2 Results FDA Drug

• 1 other identifier: SPIMM-202
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 4

Brief 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.

Conditions

Primary Mitochondrial Disease

Keywords

Primary Mitochondrial Disease; Mitochondrial Myopathy; Elamipretide; MTP-131; Bendavia™

Outcome Measures

Primary Outcomes (1)

• Distance Walked on the 6-minute Walk Test (6MWT) by Visit

  Distance in meters walked on the 6-minute walk test (6MWT) at end of treatment, where end of treatment means end of week 4 for Period 1 and end of week 12 for Period 2.

  End of Week 4 and End of Week 12

Secondary Outcomes (9)

• Wrist Accelerometer Counts by Day

  Last 7 days prior to the date of end of treatment visit

• Average Hip Accelerator Counts by Day

  Last 7 days prior to the date of end of treatment visit

• Neuro-QoL Fatigue Short Form Score: Total T-Scores (Question 1-8)

  End of Week 4 and End of Week 12

• Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) Total Fatigue Score by Week

  Last 7 days of Week 4 and Last 7 days of Week 12

• Triple Timed Up and Go (3TUG) Test by Visit

  End of Week 4 and End of Week 12

Study Arms (2)

Elamipretide, Then Placebo

EXPERIMENTAL

Participants first received 40 mg of elamipretide once daily subcutaneously for 4 weeks. After a washout period of 4 weeks, they then received placebo administered once daily subcutaneously for 4 weeks.

Drug: Elamipretide; Drug: Placebo

Placebo, Then Elamipretide

PLACEBO COMPARATOR

Participants first received placebo once daily subcutaneously for 4 weeks. After a washout period of 4 weeks, they then received 40 mg of elamipretide once daily subcutaneously for 4 weeks.

Drug: Elamipretide; Drug: Placebo

Interventions

Elamipretide DRUG

4 weeks of treatment with 40 mg elamipretide administered once daily subcutaneously

Also known as: Bendavia, MTP-131

Elamipretide, Then Placebo; Placebo, Then Elamipretide

Placebo DRUG

4 weeks of treatment with placebo administered once daily subcutaneously

Elamipretide, Then Placebo; Placebo, Then Elamipretide

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Stealth BioTherapeutics Inc.: lead

Study Sites (4)

University of California

San Diego, California, 92093, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Akron Children's Hospital

Akron, Ohio, 44308, United States

Location

Children's Hospital of Pittsburg of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Related Publications (1)

• Gwaltney C, Stokes J, Aiudi A, Mazar I, Ollis S, Love E, Karaa A, Houts CR, Wirth RJ, Shields AL. Psychometric performance of the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA) in a randomized, double-blind, placebo-controlled crossover study in subjects with mitochondrial disease. J Patient Rep Outcomes. 2022 Dec 23;6(1):129. doi: 10.1186/s41687-022-00534-y.

  PMID: 36562873 DERIVED

MeSH Terms

Conditions

Mitochondrial Myopathies

Interventions

arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Rekha Sathyanarayana, Executive Director, Clinical Operations
• Organization: Stealth BioTherapeutics Inc.

Rekha.Sathyanarayana@stealthbt.com

617-762-2579

Study Officials

• Jim Carr

  Stealth BioTherapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 13, 2016
• First Posted: June 20, 2016
• Study Start: August 22, 2016
• Primary Completion: March 10, 2017
• Study Completion: March 23, 2017
• Last Updated: July 17, 2020
• Results First Posted: July 7, 2020

Record last verified: 2020-07

Locations

US (4)