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History of Changes for Study: NCT03098797
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome (TAZPOWER)
Latest version (submitted March 7, 2022) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 March 27, 2017 None (earliest Version on record)
2 April 18, 2017 Study Status and Contacts/Locations
3 May 8, 2017 Recruitment Status, Study Status, Contacts/Locations and Oversight
4 October 26, 2017 Study Status and Contacts/Locations
5 March 6, 2018 Recruitment Status, Study Status, Contacts/Locations and Study Identification
6 September 4, 2018 Study Status
7 October 4, 2018 Study Status
8 February 13, 2019 Study Status
9 January 24, 2020 Study Status
10 April 10, 2020 Study Status and Study Design
11 November 5, 2021 Study Status
12 March 7, 2022 Recruitment Status and Study Status
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Study NCT03098797
Submitted Date:  March 27, 2017 (v1)

Open or close this module Study Identification
Unique Protocol ID: SPIBA-201
Brief Title: A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome (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
Secondary IDs:
Open or close this module Study Status
Record Verification: March 2017
Overall Status: Not yet recruiting
Study Start: March 2017
Primary Completion: August 2018 [Anticipated]
Study Completion: October 2018 [Anticipated]
First Submitted: March 21, 2017
First Submitted that
Met QC Criteria:
March 27, 2017
First Posted: April 4, 2017 [Actual]
Last Update Submitted that
Met QC Criteria:
March 27, 2017
Last Update Posted: April 4, 2017 [Actual]
Open or close this module Sponsor/Collaborators
Sponsor: Stealth BioTherapeutics Inc.
Responsible Party: Sponsor
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug: Yes
U.S. FDA-regulated Device: No
Data Monitoring: Yes
Open or close this module 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.
Detailed Description:
Open or close this module Conditions
Conditions: Barth Syndrome
Keywords: Barth Syndrome
elamipretide
MTP-131
Stealth
Stealth BT
BTHS
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 2/Phase 3
Interventional Study Model: Crossover Assignment
Number of Arms: 2
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Allocation: Randomized
Enrollment: 12 [Anticipated]
Open or close this module Arms and Interventions
Arms Assigned Interventions
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 Names:
  • 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 Names:
  • MTP-131
Open or close this module Outcome Measures
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 ]

Open or close this module Eligibility
Minimum Age: 12 Years
Maximum Age:
Sex: Male
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

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
Open or close this module Contacts/Locations
Locations: United States, Maryland
McKusick-Nathans Institute of Generic Medicine, Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21287
Contact:Contact: Hilary Vernon, MD, PhD 410-502-8625 hvernon@jhmi.edu
Open or close this module IPDSharing
Plan to Share IPD: Undecided
Open or close this module References
Citations:
Links:
Available IPD/Information:

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