Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Dise… (NCT05162768) | Clinical Trial Compass
CompletedPhase 3
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)
United States102 participantsStarted 2022-04-29
Plain-language summary
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 mutations (nPMD).
Who can participate
Age range18 Years – 70 Years
SexALL
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Inclusion criteria
✓. Willing and able to provide a signed informed consent form (ICF) prior to participation in any trial-related procedures.
✓. Agrees and is able to adhere to the trial requirements for the length of the trial, including administration of assigned treatment.
✓. Is ≥18 years and ≤ 70 years of age at the time of screening.
✓. Diagnosed with nPMD with a predominant clinical manifestation of myopathy, which must include progressive external ophthalmoplegia (PEO) and exercise intolerance and/or skeletal muscle weakness, with genetic confirmation of either:
✓. Nuclear DNA mutation of the mitochondrial replisome (replisome-related mutations), which include the following genes:
✓. Other pathogenic mutations specific to nuclear DNA.
✓. Women of childbearing potential must agree to use one of the following methods of birth control from the date they sign the ICF until 28 days after the last dose of IMP:
✓. Abstinence, when it is in line with the preferred and usual lifestyle of the subject. Subject agrees to use a highly effective method of contraception should they become sexually active.
Exclusion criteria
✕. Is unable to perform the 6MWT, 3TUG, or 5XSST functional tests. The use of a gait assist device is allowed; however, use should remain consistent for the entire duration of the trial.
✕. Female subjects who are pregnant, planning to become pregnant, or breastfeeding/lactating.
✕. Walks \< 150 meters or \> 450 meters during the 6MWT (Screening Visit only).
✕. The estimated glomerular filtration rate (eGFR) is \< 30 mL/min/1.73 m2, using the Modification of Diet in Renal Disease (MDRD) Study equation (Screening Visit only).
✕. Has undergone an in-patient hospitalization within 30 days prior to screening or has a planned hospitalization or a surgical procedure during the trial, unless, in the opinion of the Investigator, it is concluded that it will not impact the outcome measurements of the trial.
✕. Has clinically significant respiratory disease and/or cardiac disease that would interfere with trial assessments, in the opinion of the Investigator.
✕. Has had any prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to screening.
✕. Has history of or current severe neurologic impairment, severe epilepsy, severe ataxia, or severe neuropathy that may interfere with their ability to complete all trial requirements, in the opinion of the Investigator.