Primary Mitochondrial Myopathy Clinical Trial
Official title:
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
The purpose of the phase 2 portion of this study is to select a biologically-active ASP0367 dose level by pharmacokinetic (PK) and pharmacodynamic (PD) evaluation. The phase 2 portion of this study will also assess the safety and tolerability of ASP0367. The purpose of the phase 3 portion of this study is to assess the effect of ASP0367 on functional improvement relative to placebo and will also assess the safety and tolerability of ASP0367 relative to placebo. The phase 3 portion of this study will also assess the effect of ASP0367 on functional improvement and fatigue relative to placebo and will assess the effect of ASP0367 in overall participant functioning relative to placebo.
Efficacy (i.e., functional improvement) will be assessed by a functional motor test, 6-minute walk test (6MWT). The study consists of the following portions: screening (4 weeks); phase 2 dose selection portion with 2 doses of ASP0367 versus matching placebo (2 weeks); phase 3 portion with selected, single dose treatment versus placebo (up to 52 weeks); open-label extension (OLE) (24 weeks); and follow-up (4 weeks). During the phase 2 portion of the study, participants will be randomly placed into 1 of 3 arms (low dose ASP0367, high dose ASP0367 or placebo). The relevant dose level will be selected based on the pharmacokinetics/pharmacodynamics and overall safety. Participants will maintain their original Phase 2 dose level and no new participants will be enrolled in the study until phase 3 dose selection is made. After the phase 3 dose is selected, all participants except the placebo group will switch to the selected dose level of ASP0367 for the remainder of the phase 3 portion of the study (up to a total 52 weeks including the phase 2 portion). Participants who were originally assigned to placebo will remain on placebo for up to 52 weeks. The remaining enrollment of participants will be randomized to either ASP0367 or matching placebo at a ratio of 1:1. All participants who have completed the phase 3 portion of the study and are eligible for the OLE will be offered the opportunity to take ASP0367 for an additional 24 weeks. ;
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