Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) (NuPower)

  • End date
    Jun 14, 2024
  • participants needed
  • sponsor
    Stealth BioTherapeutics Inc.
Updated on 7 October 2022


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).


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.

Condition Mitochondrial Myopathies, Mitochondrial Pathology, Mitochondrial DNA Mutation, Mitochondrial Diseases, Mitochondrial DNA Deletion, Mitochondrial DNA Depletion, Mitochondrial Metabolism Defect, Mitochondrial Complex I Deficiency
Treatment Placebo, elamipretide
Clinical Study IdentifierNCT05162768
SponsorStealth BioTherapeutics Inc.
Last Modified on7 October 2022


Yes No Not Sure

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: 1\. Willing and able to provide a signed informed
consent form (ICF) prior to participation in any trial-related procedures. 2\
Agrees and is able to adhere to the trial requirements for the length of the
trial, including administration of assigned treatment. 3\. Is ≥18 years and ≤
years of age at the time of screening. 4\. 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: 1\. Nuclear DNA mutation of the
mitochondrial replisome (replisome-related mutations), which include the
following genes: \- POLG 1/2 \- TWINKLE (C10ORF2) \- TYMP \- DGUOK \- TK2 \-
RRM2B \- RNASEH1 \- SSBP \- MGME1 \- DNA2 \- ANT1 (SLC25A4) \- SUCLG1 \-
SUCLA2 \- MPV17 or 2\. Other pathogenic mutations specific to nuclear DNA. 5\
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: 1\. 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. 2\. Relationships with male
partners who have been surgically sterilized by vasectomy (the vasectomy
procedure must have been conducted at least 60 days prior to the Screening
Visit). 3\. Barrier method (e.g., condom or occlusive cap) with spermicidal
injectable) or an intrauterine device or system. 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). 6\. 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
foam/gel/film/cream AND either hormonal contraception (oral, implanted, or

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. 2\. Female
subjects who are pregnant, planning to become pregnant, or
breastfeeding/lactating. 3\. Walks < 150 meters or > 450 meters during the
MWT (Screening Visit only). 4\. 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). 5\. 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. 6\. Has clinically significant respiratory
disease and/or cardiac disease that would interfere with trial assessments, in
the opinion of the Investigator. 7\. 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. 9\
Active malignancy or any other cancer from which the subject has been disease-
free for < 2 years. Localized squamous or non-invasive basal cell skin
carcinomas are allowed, if appropriately treated prior to screening. 10\. Has
had a solid organ transplant. 11\. Has been previously diagnosed with human
immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection. 12\. Has
a history of a systemic eosinophilic illness and/or an eosinophil count >1,000
cells x106/L at the Screening Visit. 13\. Is currently participating or has
participated in an interventional clinical trial (i.e., investigational
product or device, stem cell therapy, gene therapy) within 30 days prior to
current trial; or is currently enrolled in a non-interventional clinical trial
that, in the opinion of the Investigator, may be potentially confounding to
the results of the current trial (e.g., exercise therapy trial). 14\. Has
received elamipretide (MTP-131) within the past one year of the Screening
Visit. 15\. Has a history of active substance abuse during the year prior, in
the opinion of the Investigator
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