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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.
November 18, 2016
November 29, 2016
March 3, 2017
December 2016
December 2021   (Final data collection date for primary outcome measure)
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
Same as current
Complete list of historical versions of study NCT02976038 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Disease (PMD)
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)
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
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.
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Primary Mitochondrial Disease
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.
Experimental: elamipretide
Open-label once daily subcutaneous injection of 40mg elamipretide
Intervention: Drug: elamipretide
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Enrolling by invitation
36
December 2021
December 2021   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
16 Years and older   (Child, Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT02976038
SPIMM-203
No
Not Provided
Plan to Share IPD: No
Stealth BioTherapeutics Inc.
Stealth BioTherapeutics Inc.
Not Provided
Not Provided
Stealth BioTherapeutics Inc.
March 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP