SPIMD-301 is a 48-week, randomized, double-blind, parallel-group, placebo-controlled trial to
assess efficacy and safety of single daily subcutaneous (SC) administration of elamipretide
as a treatment for subjects with primary mitochondrial myopathy associated with nuclear DNA
This 48-week randomized, double-blind, parallel-group, placebo-controlled trial will enroll
approximately 130 subjects, consisting of 90 subjects who have nPMD associated with
pathogenic mutations of the mitochondrial replisome("replisome-related mutations") for
primary analysis and an additional subset of up to 40 subjects who have nPMD associated with
other non-replisome-related pathogenic mutations specific to the nuclear DNA. Efficacy and
safety of single daily SC doses of elamipretide administered as a treatment for subjects who
have primary mitochondrial myopathy associated with nPMD will be determined. Subjects will be
randomized 1:1 to 60mg Elamipretide or matching placebo groups.
Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases, Mitochondrial DNA Deletion, Mitochondrial DNA Depletion, Mitochondrial Metabolism Defect, Mitochondrial Complex I Deficiency
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.