Primary Mitochondrial Disease Clinical Trial
Official title:
A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Study to Evaluate Safety, Tolerability, and Efficacy of Subcutaneous Injections of MTP-131 in Subjects With Mitochondrial Myopathy Previously Treated in the SPIMM-201 Study
This randomized, double-blind, placebo-controlled, crossover study screened 32 subjects with primary mitochondrial myopathy (PMM) to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of subcutaneous elamipretide in this patient population.
This randomized, double-blind, placebo-controlled, crossover study screened 32 subjects with
primary mitochondrial myopathy (PMM) who had completed participation in the SPIMM-201 study
where they had received 5 days of intravenous (IV) elamipretide (0.01, 0.10, or 0.25
mg/kg/hour infused for 2 hours) or placebo (randomized 3:1). The primary objective was to
evaluate the effect of single daily subcutaneous (SC) doses of elamipretide administered for
4 weeks on the 6-minute walking distance (6MWD).
Subjects were randomized (1:1) to one of two sequence groups: 4-weeks of treatment with 40 mg
elamipretide administered once daily SC in Treatment Period 1 followed by 4-weeks of
treatment with placebo administered once daily SC in Treatment Period 2 (separated by a
4-week washout period), or vice versa. Each sequence group went through 5 distinct periods:
Screening, Treatment Period 1, Washout, Treatment Period 2, and Follow-Up. Safety,
tolerability, pharmacokinetics (PK), and efficacy of subcutaneous elamipretide in this
patient population were analyzed.
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|---|---|---|---|
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