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A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome (TAZPOWER)

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ClinicalTrials.gov Identifier: NCT03098797
Recruitment Status : Recruiting
First Posted : April 4, 2017
Last Update Posted : October 27, 2017
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.

Brief Summary:
A randomized double-blind cross over trial to evaluate the safety, efficacy and tolerability of elamipretide in subjects with Barth Syndrome.

Condition or disease Intervention/treatment Phase
Barth Syndrome Drug: Elamipretide Phase 2 Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Crossover Trial to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Barth Syndrome
Actual Study Start Date : May 1, 2017
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : October 2018

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: Experimental: Elamipretide
Patients will be randomized to receive 12 weeks of elamipretide in one of the two treatment periods. All subjects will receive 12 weeks of elamipretide and 12 weeks of placebo.
Drug: Elamipretide
40 mg daily subcutaneous injection for 12 weeks
Other Name: MTP-131
Placebo Comparator: Placebo
Patients will be randomized to receive 12 weeks of placebo in one of the two treatment periods. All subjects will receive 12 weeks of placebo and 12 weeks of elamipretide.
Drug: Elamipretide
40 mg daily subcutaneous injection for 12 weeks
Other Name: MTP-131

Primary Outcome Measures :
  1. Change in distance walked during the 6-minute walk test (6MWT) [ Time Frame: 12 weeks ]

Secondary Outcome Measures :
  1. Change in muscle strength as measured by handheld dynamometry [ Time Frame: 12 weeks ]
  2. Change in five times sit-to-stand test [ Time Frame: 12 weeks ]
  3. Change in 2-D and 3-D echocardiographic measurements [ Time Frame: 12 weeks ]
  4. Change in accelerometry counts [ Time Frame: 12 weeks ]
  5. Change in SWAY application balance assessments [ Time Frame: 12 weeks ]
  6. Change in Patient Reported Outcomes [ Time Frame: 12 weeks ]
  7. Change in Clinician Global Impression [ Time Frame: 12 weeks ]
  8. Change in biomarkers [ Time Frame: 12 weeks ]
  9. Number of participants with treatment related adverse events [ Time Frame: 12 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Genetically confirmed Barth Syndrome
  • Male aged 12 and above
  • At the screening visit, eGFR must meet the following:

    1. Body weight >30 kg AND eGFR > 90mL/min at screening
    2. Body weight >40kg AND eGFR >60 but <90mL/min at screening
  • Ambulatory and impaired during the 6MWT
  • On stable medication for 30 days prior to the baseline visit

Exclusion Criteria:

  • Participated in another interventional clinical trial within 30 days of or is currently enrolled in a non-interventional clinical trial at the baseline visit potentially confounding with this trial
  • Prior or current medical condition that would prevent the subject from safely participating in the trial
  • Undergone any inpatient hospitalizations within 30 days of the baseline visit
  • Is undergoing an apparent pubertal growth spurt
  • Has uncontrolled hypertension
  • History of substance abused within the year before the baseline visit or is likely to be uncompliant
  • History of heart transplantation or current placement on the waiting list for a heart transplant
  • For subjects with an ICD: known occurrance of ICD discharge in the 3 months prior to the baseline visit
  • For subjects without an ICD: expected to undergo an implantation of an ICD during the conduct of the study
  • Currently receiving treatment with chemotherapeutic agents or immunosuppressant agents or has received prior radiation therapy to the chest
  • Recipient of stem cell or gene therapy or is currently being treated by a therapeutic investigational device

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): NCT03098797

United States, Maryland
McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine Recruiting
Baltimore, Maryland, United States, 21287
Contact: Hilary Vernon, MD, PhD    410-502-8625    hvernon1@jhmi.edu   
Sponsors and Collaborators
Stealth BioTherapeutics Inc.

Responsible Party: Stealth BioTherapeutics Inc.
ClinicalTrials.gov Identifier: NCT03098797     History of Changes
Other Study ID Numbers: SPIBA-201
First Posted: April 4, 2017    Key Record Dates
Last Update Posted: October 27, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Stealth BioTherapeutics Inc.:
Barth Syndrome
Stealth BT

Additional relevant MeSH terms:
Barth Syndrome
Pathologic Processes
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases