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

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.
 
ClinicalTrials.gov Identifier: NCT03098797
Recruitment Status : Completed
First Posted : April 4, 2017
Last Update Posted : March 9, 2022
Sponsor:
Information provided by (Responsible Party):
Stealth BioTherapeutics Inc.

Tracking Information
First Submitted Date  ICMJE March 21, 2017
First Posted Date  ICMJE April 4, 2017
Last Update Posted Date March 9, 2022
Actual Study Start Date  ICMJE May 1, 2017
Actual Primary Completion Date October 11, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 27, 2017)
Change in distance walked during the 6-minute walk test (6MWT) [ Time Frame: 12 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 27, 2017)
  • Change in muscle strength as measured by handheld dynamometry [ Time Frame: 12 weeks ]
  • Change in five times sit-to-stand test [ Time Frame: 12 weeks ]
  • Change in 2-D and 3-D echocardiographic measurements [ Time Frame: 12 weeks ]
  • Change in accelerometry counts [ Time Frame: 12 weeks ]
  • Change in SWAY application balance assessments [ Time Frame: 12 weeks ]
  • Change in Patient Reported Outcomes [ Time Frame: 12 weeks ]
  • Change in Clinician Global Impression [ Time Frame: 12 weeks ]
  • Change in biomarkers [ Time Frame: 12 weeks ]
  • Number of participants with treatment related adverse events [ Time Frame: 12 weeks ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
Official Title  ICMJE 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 Followed by an Open-Label Treatment Extension
Brief Summary A randomized double-blind cross over trial to evaluate the safety, efficacy and tolerability of elamipretide in subjects with Barth Syndrome.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Intervention Model Description:
28 wks, double-blinded cross over followed by 168 wks open label long term safety & tolerability trial.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Barth Syndrome
Intervention  ICMJE Drug: Elamipretide
40 mg daily subcutaneous injection for 12 weeks
Other Name: MTP-131
Study Arms  ICMJE
  • 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.
    Intervention: Drug: Elamipretide
  • 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.
    Intervention: Drug: Elamipretide
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 27, 2017)
12
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 2, 2021
Actual Primary Completion Date October 11, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03098797
Other Study ID Numbers  ICMJE SPIBA-201
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Current Responsible Party Stealth BioTherapeutics Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Stealth BioTherapeutics Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Stealth BioTherapeutics Inc.
Verification Date March 2022

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