Children's Hospital Colorado | Pediatric Infectious Disease
Status and phase
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About
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).
Full description
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.
Enrollment
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Volunteers
Inclusion criteria
A subject must meet all of the following inclusion criteria at the Screening and Baseline Visit (unless otherwise specified) to be eligible for inclusion in the SPIMD-301 trial:
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:
Note: Non-childbearing potential is defined as surgical sterilization (e.g., bilateral oophorectomy, hysterectomy, or tubal ligation) or postmenopausal (defined as permanent cessation of menstruation for at least 12 consecutive months prior to the Screening Visit).
Male subjects with female partners of childbearing potential must be willing to use a highly effective method of contraception from the date they sign the ICF until 28 days after the last dose of IMP.
Exclusion criteria
Primary purpose
Allocation
Interventional model
Masking
102 participants in 2 patient groups, including a placebo group
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Central trial contact
Rekha Sathyanarayana
Data sourced from clinicaltrials.gov
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