NCT03323749

A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension

A Phase 3 Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 218 (actual)
• Sponsor: Stealth BioTherapeutics Inc. · Industry
• Sex: All
• Age: 16 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter phase 3 randomized, double-blind, parallel-group, placebo-controlled trial to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial myopathy. This will be followed by an open-label treatment extension.

Detailed description

Part 11 is a 24-week, randomized, double-blind, parallel-group, placebo-controlled assessment of the efficacy and safety of single daily subcutaneous (SC) doses of 40 mg elamipretide (vs placebo) administered with the elamipretide delivery system as a treatment for subjects with primary mitochondrial myopathy (PMM). Part 2 was to assess the long-term safety and tolerability of single daily SC doses of 40 mg elamipretide administered with the elamipretide delivery system for up to 144 weeks.

Conditions

• Primary Mitochondrial Myopathy

Interventions

Type	Name	Description
COMBINATION_PRODUCT	elamipretide	40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
COMBINATION_PRODUCT	placebo comparator	40 mg of placebo administered as once daily 0.5 mL subcutaneous injections for 24 weeks using the elamipretide delivery system
COMBINATION_PRODUCT	elamipretide open label treatment	40 mg of elamipretide administered as once daily 0.5 mL subcutaneous injections for up to 144 weeks using the elamipretide delivery system

Timeline

• Start date: 2017-10-09
• Primary completion: 2020-02-10
• Completion: 2020-02-10
• First posted: 2017-10-27
• Last updated: 2022-01-24
• Results posted: 2021-04-02

Locations

27 sites across 7 countries: United States, Canada, Denmark, Germany, Hungary, Italy, United Kingdom

Regulatory

• FDA-regulated drug study