At a glance
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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
In Brief
A Phase 2 clinical trial evaluating elamipretide (low dose), elamipretide (intermediate dose), and 2 other interventions for Mitochondrial Myopathy. Completed, enrolled 36 participants across 4 sites.
Detailed Summary
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.
Study Details
Timeline
Interventions
elamipretide (0.01 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days
elamipretide (0.10 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days
elamipretide (0.25 mg/kg/hr) administered as single day intravenous infusion over 2 hours for 5 days
placebo (at each dose cohort) administered as single day intravenous infusion over 2 hours for 5 days