Clinical Trial: 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
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
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 Genetica
Brief Summary: 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
Detailed Summary:
Sponsor: Stealth BioTherapeutics Inc.
Current Primary Outcome: Distance walked (meters) on the 6-minute walk test (6MWT) [ Time Frame: 4 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- 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 ]
Original Secondary Outcome: Same as current
Information By: Stealth BioTherapeutics Inc.
Dates:
Date Received: June 13, 2016
Date Started: July 2016
Date Completion: May 2017
Last Updated: January 23, 2017
Last Verified: January 2017