Back to resultsA Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome (TAZPOWER)
Primary Purpose
Barth Syndrome
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Elamipretide
Sponsored by
About this trial
This is an interventional treatment trial for Barth Syndrome focused on measuring Barth Syndrome, elamipretide, MTP-131, Stealth, Stealth BT, BTHS
Eligibility Criteria
Inclusion Criteria:
- Genetically confirmed Barth Syndrome
- Male aged 12 and above
At the screening visit, eGFR must meet the following:
- Body weight >30 kg AND eGFR > 90mL/min at screening
- 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
Sites / Locations
- McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Experimental: Elamipretide
Placebo
Arm Description
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.
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.
Outcomes
Primary Outcome Measures
Change in distance walked during the 6-minute walk test (6MWT)
Secondary Outcome Measures
Change in muscle strength as measured by handheld dynamometry
Change in five times sit-to-stand test
Change in 2-D and 3-D echocardiographic measurements
Change in accelerometry counts
Change in SWAY application balance assessments
Change in Patient Reported Outcomes
Change in Clinician Global Impression
Change in biomarkers
Number of participants with treatment related adverse events
Full Information
NCT ID
NCT03098797
First Posted
March 21, 2017
Last Updated
March 7, 2022
Sponsor
Stealth BioTherapeutics Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03098797
Brief Title
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
Acronym
TAZPOWER
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 Followed by an Open-Label Treatment Extension
Study Type
Interventional
2. Study Status
Record Verification Date
March 2022
Overall Recruitment Status
Completed
Study Start Date
May 1, 2017 (Actual)
Primary Completion Date
October 11, 2021 (Actual)
Study Completion Date
December 2, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Stealth BioTherapeutics Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
A randomized double-blind cross over trial to evaluate the safety, efficacy and tolerability of elamipretide in subjects with Barth Syndrome.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Barth Syndrome
Keywords
Barth Syndrome, elamipretide, MTP-131, Stealth, Stealth BT, BTHS
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Crossover Assignment
Model Description
28 wks, double-blinded cross over followed by 168 wks open label long term safety & tolerability trial.
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Experimental: Elamipretide
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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 Type
Drug
Intervention Name(s)
Elamipretide
Other Intervention Name(s)
MTP-131
Intervention Description
40 mg daily subcutaneous injection for 12 weeks
Primary Outcome Measure Information:
Title
Change in distance walked during the 6-minute walk test (6MWT)
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Change in muscle strength as measured by handheld dynamometry
Time Frame
12 weeks
Title
Change in five times sit-to-stand test
Time Frame
12 weeks
Title
Change in 2-D and 3-D echocardiographic measurements
Time Frame
12 weeks
Title
Change in accelerometry counts
Time Frame
12 weeks
Title
Change in SWAY application balance assessments
Time Frame
12 weeks
Title
Change in Patient Reported Outcomes
Time Frame
12 weeks
Title
Change in Clinician Global Impression
Time Frame
12 weeks
Title
Change in biomarkers
Time Frame
12 weeks
Title
Number of participants with treatment related adverse events
Time Frame
12 weeks
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Genetically confirmed Barth Syndrome
Male aged 12 and above
At the screening visit, eGFR must meet the following:
Body weight >30 kg AND eGFR > 90mL/min at screening
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
Facility Information:
Facility Name
McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
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