We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

This study is enrolling participants by invitation only.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02976038
First Posted: November 29, 2016
Last Update Posted: March 3, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.
  Purpose
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

Condition Intervention Phase
Primary Mitochondrial Disease Drug: elamipretide Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Disease (PMD)

Resource links provided by NLM:


Further study details as provided by Stealth BioTherapeutics Inc.:

Primary Outcome Measures:
  • Incidence of adverse events [ Time Frame: Baseline through end of study for up to 260 weeks ]
    Assessing long-term safety and tolerability through collection of adverse events


Secondary Outcome Measures:
  • 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 Triple Timed up and Go Test [ 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 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 the NeuroQOL Fatigue Questionnaire [ Time Frame: up to 260 weeks ]
  • Changes in the Patient Global Assessment [ Time Frame: up to 260 Weeks ]
  • Changes in Primary Mitochondrial Disease Symptom Assessment [ Time Frame: Up to 260 weeks ]
  • Changes in Work Limitations Questionnaire [ Time Frame: Up to 260 Weeks ]
  • Changes in Physician Global Assessment [ Time Frame: Up to 260 Weeks ]

Estimated Enrollment: 36
Actual Study Start Date: December 2016
Estimated Study Completion Date: December 2021
Estimated Primary Completion Date: December 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: elamipretide
Open-label once daily subcutaneous injection of 40mg elamipretide
Drug: elamipretide
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.

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02976038


Locations
United States, California
University of California
San Diego, California, United States, 92123
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
United States, Ohio
Akron Children's Hospital
Akron, Ohio, United States
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Sponsors and Collaborators
Stealth BioTherapeutics Inc.
  More Information

Responsible Party: Stealth BioTherapeutics Inc.
ClinicalTrials.gov Identifier: NCT02976038     History of Changes
Other Study ID Numbers: SPIMM-203
First Submitted: November 18, 2016
First Posted: November 29, 2016
Last Update Posted: March 3, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Stealth BioTherapeutics Inc.:
Primary Mitochondrial Disease
Stealth
elamipretide

Additional relevant MeSH terms:
Mitochondrial Diseases
Metabolic Diseases