Mitochondrial Myopathy Clinical Trial
Official title:
Phase 1/2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Clinical Study for the Safety, Tolerability, and Efficacy of IV MTP-131 for Mitochondrial Myopathy in Genetically Confirmed Mitochondrial Disease
Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of MTP-131 in this patient population.
This multi-center, randomized, double-blind, placebo-controlled study enrolled 36 subjects
into 3 cohorts of 12 subjects each to evaluate treatment with 3 ascending doses of
intravenous elamipretide (0.01, 0.10, and 0.25 mg/kg/hr infused for 2 hours). After each
cohort, a Safety Monitoring Board (SMB) determined if dose escalation to the next higher dose
of elamipretide was warranted. Each cohort went through 3 distinct periods: Screening,
Treatment, and Follow-up.
The Screening Period started with informed consent and may have lasted up to 40 days. During
this period, screening procedures to determine subject eligibility for the study occurred,
including confirmation of disease, which incorporated a committee review of the
investigator-submitted diagnosis and genetic results. The Treatment Period began on Day 1
(Visit 2) and lasted for 5 days (until Day 5 [Visit 6]). Within each cohort, 9 subjects were
randomized to active drug and 3 subjects were randomized to placebo on Day 1 and subjects
received treatment once a day for 5 consecutive days. Safety, tolerability, and efficacy
measures were performed at pre-specified times. The Follow-up Period began at the time of
discharge on Day 5. Subjects returned to the study center for the Follow-up Visit on Day 7
(+1 day).
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