NCT04641962

A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy

A Randomized, Double-blind, Placebo-controlled Phase 2 Study to Assess the Efficacy, Safety and Tolerability of ASP0367 in Participants With Primary Mitochondrial Myopathy

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 34 (actual)
• Sponsor: Astellas Pharma Inc · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to evaluate the dose response of Bocidelpar on functional improvement relative to placebo, safety, and tolerability in participants with Primary Mitochondrial Myopathy.

Detailed description

Efficacy (i.e., functional improvement) was assessed by a functional motor test, 6-minute walk test (6MWT). The study consisted of the following portions: screening (4 weeks); double-blind treatment period with 2 doses of Bocidelpar vs matching placebo (24 weeks) and follow up (4 weeks). Participants were randomly placed into 1 of 3 arms (Bocidelpar 30 mgs, Bocidelpar 75 mgs or placebo).

Conditions

• Primary Mitochondrial Myopathy

Interventions

Type	Name	Description
DRUG	Bocidelpar	Oral
DRUG	Placebo	Oral

Timeline

• Start date: 2021-05-24
• Primary completion: 2024-05-08
• Completion: 2024-05-08
• First posted: 2020-11-24
• Last updated: 2025-07-03
• Results posted: 2025-06-17

Locations

11 sites across 1 country: United States

Regulatory

• FDA-regulated drug study