Current Research Studies
A Randomized, Double-blind, Placebo-controlled Adaptive Phase 2/3 Study with Open-label Extension to Assess the Efficacy, Safety and Tolerability of ASP0367 in Participants with Primary Mitochondrial Myopathy
Astellas_PMM
What is the goal of the study?
Study Design Overview: This is a randomized, double-blind, placebo-controlled, oral dose, adaptive phase 2/3 study with open-label extension (OLE) to evaluate the efficacy, safety and tolerability of ASP0367 in participants with PMM. Efficacy (i.e., functional improvement) will be assessed by a functional motor test, 6MWT. The study consists of the following portions: screening (4 weeks); phase 2 dose selection portion with 2 doses of ASP0367 vs matching placebo (2 weeks); phase 3 portion with selected, single dose treatment vs placebo (up to 52 weeks); OLE (24 weeks); and follow-up (4 weeks).
Who can participate in the study?
Please contact the study team listed below to learn more.