Clinical Trial: A Study Investigating the Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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 Treat

Brief Summary: 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.

Detailed Summary:
Sponsor: Stealth BioTherapeutics Inc.

Current Primary Outcome:

  • 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 ]


Original Primary Outcome:

  • 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 ]


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

Original Secondary Outcome:

  • Change from Baseline in distance walked (meters) on the 6-minute walk test (6MWT) [ Time Frame: Assessed at Day 5 (end-of-treatment visit) and Day 7 ]
  • Change from Baseline in selected outputs of Cardiopulmonary Exercise Testing (CPET) [ Time Frame: Assessed at Day 5 (end-of-treatment visit) and Day 7 ]


Information By: Stealth BioTherapeutics Inc.

Dates:
Date Received: February 9, 2015
Date Started: February 2015
Date Completion:
Last Updated: May 27, 2016
Last Verified: May 2016