Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02976038
• Date of registration: 18/11/2016
• Prospective Registration: Yes
• Primary sponsor: Stealth BioTherapeutics Inc.
• Public title: Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)
• Scientific title: A Multicenter, Open-Label Phase 2 Extension Trial to Characterize the Long-term Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)
• Date of first enrolment: December 2016
• Target sample size: 36
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02976038
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Investigator determines the subject can, and subject agrees to, adhere to the trial
requirements for the length of the trial including self-administration (by subject or
trained caregiver) of the study drug

- Subject completed the End-of-Study Visit in SPIMM-202

Exclusion Criteria:

- Subject has any prior or current medical condition that, in the judgment of the
Investigator, would prevent the subject from safely participating in and/or completing
all trial requirements

- Subject has received any investigational compound (excluding elamipretide) and/or has
participated in another interventional clinical trial within 30 days prior to the
SPIMM-203 Baseline Visit (excluding SPIMM-202) or is concurrently enrolled in any
non-interventional research of any type judged to be scientifically or medically
incompatible with the trial as deemed by the Investigator in consultation with the
Sponsor

- Subject experienced an adverse reaction attributed to study drug resulting in
permanent discontinuation of study drug in the SPIMM-202 trial.

- Female subjects who are pregnant, planning to become pregnant, or lactating

- Subject has undergone an in-patient hospitalization within the 1 month prior to the
SPIMM-203 Baseline Visit



Age minimum: 16 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primary Mitochondrial Disease

Intervention(s)

Drug: elamipretide

Primary Outcome(s)

Incidence of adverse events [Time Frame: Baseline through end of study for up to 260 weeks]

Secondary Outcome(s)

Changes in Work Limitations Questionnaire [Time Frame: Up to 260 Weeks]

Changes in Physician Global Assessment [Time Frame: Up to 260 Weeks]

Changes in Scale for the Assessment and Rating of Ataxia [Time Frame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 260 weeks]

Changes in 5X Sit to Stand [Time Frame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 260 weeks]

Changes in distance walked on Six Minute Walk Test [Time Frame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 260 weeks]

Changes in the NeuroQOL Fatigue Questionnaire [Time Frame: up to 260 weeks]

Changes in the Patient Global Assessment [Time Frame: up to 260 Weeks]

Changes in Triple Timed up and Go Test [Time Frame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 260 weeks]

Changes in Primary Mitochondrial Disease Symptom Assessment [Time Frame: Up to 260 weeks]

Secondary ID(s)

SPIMM-203

Source(s) of Monetary Support

Please refer to primary and secondary sponsors