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

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

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.

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

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 12 participants
• 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
• 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
• Actual Study Start Date: May 1, 2017
• Actual Primary Completion Date: October 11, 2021
• Actual Study Completion Date: December 2, 2021

Arms and Interventions

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

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 ]

Eligibility Criteria

• Ages Eligible for Study: 12 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: Male
• 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

Contacts and Locations

Locations

United States, Maryland

McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine

Baltimore, Maryland, United States, 21287

Sponsors and Collaborators

Stealth BioTherapeutics Inc.

More Information

• Responsible Party: Stealth BioTherapeutics Inc.
• ClinicalTrials.gov Identifier: NCT03098797
• Other Study ID Numbers: SPIBA-201
• First Posted: April 4, 2017
• Last Update Posted: March 9, 2022
• Last Verified: March 2022

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; elamipretide; MTP-131; Stealth; Stealth BT; BTHS

Additional relevant MeSH terms:

Barth Syndrome; Syndrome; Disease; 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; Lipid Metabolism Disorders; Metabolic Diseases