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

NCT02976038 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: SPIMM-203
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 4

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

Conditions

Primary Mitochondrial Disease

Keywords

Primary Mitochondrial Disease; Stealth; elamipretide

Outcome Measures

Primary Outcomes (1)

• Distance Walked on Six Minute Walk Test

  Change From Baseline in Distance Walked (meters) on the Six-Minute Walk Test by Visit

  Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Secondary Outcomes (11)

• EuroQol 5 Dimensions 5 Levels (EQ-5D-5L)

  Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

• Euroquol Visual Analog Scale

  Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

• NeuroQOL Fatigue Questionnaire

  Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

• Patient Global Assessment : Categorical

  Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

• Patient Global Assessment: Continuous Variable

  Baseline, Month 3, Month 6 and Every 26 weeks for up to 160 weeks

Study Arms (1)

elamipretide

EXPERIMENTAL

Open-label once daily subcutaneous injection of 40mg elamipretide

Drug: elamipretide

Interventions

elamipretide DRUG

40 mg, subcutaneous injections (in the abdomen) daily 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.

elamipretide

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Stealth BioTherapeutics Inc.: lead

Study Sites (4)

University of California

San Diego, California, 92123, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Akron Children's Hospital

Akron, Ohio, 44308, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

MeSH Terms

Interventions

arginyl-2,'6'-dimethyltyrosyl-lysyl-phenylalaninamide

Results Point of Contact

• Title: Jim Carr, Pharm.D. Chief Clinical Development Officer
• Organization: Stealth BioTherapeutics, Inc

jim.carr@stealthbt.com

1-617-600-6888

Study Officials

• Jim Carr, PharmD

  Stealth BioTherapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2016
• First Posted: November 29, 2016
• Study Start: December 1, 2016
• Primary Completion: March 9, 2020
• Study Completion: April 9, 2020
• Last Updated: December 17, 2021
• Results First Posted: December 17, 2021

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)