Clinical trial NCT02976038: Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)
Official 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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Primary Mitochondrial Disease
Keywords: Primary Mitochondrial Disease
Stealth
elamipretide
Lead sponsor: Stealth BioTherapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2016-11-29
Enrollment: 28
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a Phase 2 Open-Label extension study to evaluate the long term safety and tolerability of daily elamipretide injections in patients with genetically confirmed Primary Mitochondrial Disease who previously participated in the SPIMM-202 Clinical Trial

Detailed description: This open-label, non-comparative, extension trial will enroll subjects with genetically confirmed PMD who have completed the End-of-Study Visit in the SPIMM-202 trial. Subjects who do not discontinue or withdraw from the trial will receive treatment with 40 mg SC elamipretide for the shortest of the following: 260 weeks; regulatory approval and commercial availability of elamipretide in the subject's respective country; or termination of the clinical development for elamipretide in subjects with PMD.

Eligibility 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

Primary outcomes:
measure: Distance Walked on Six Minute Walk Test
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
Secondary outcomes:
measure: EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
measure: Euroquol Visual Analog Scale
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
measure: NeuroQOL Fatigue Questionnaire
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
measure: Patient Global Assessment : Categorical
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
measure: Patient Global Assessment: Continuous Variable
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
measure: Work Limitations Questionnaire
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
measure: Physician Global Assessment Score (PhGA) by Visit
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
measure: Physician Global Assessment (PhGA): Categorical
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
measure: PMMSA Individual Items
timeFrame: Baseline, Week 13, 26, 52, 104, 156, End of Study (Week 160)
measure: Total Fatigue Score_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
measure: Total Fatigue Score During Activities_Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)
timeFrame: Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks
Other outcomes:
Locations:
city: San Diego
country: United States
facility: University of California
state: California
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of UPMC
state: Pennsylvania
Study officials:
affiliation: Stealth BioTherapeutics
name: Jim Carr, PharmD
role: STUDY_DIRECTOR
References: