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

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

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

Study Overview

• Status: Completed
• Conditions: Barth Syndrome
• Intervention / Treatment: Drug: Elamipretide

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / 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 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in distance walked during the 6-minute walk test (6MWT)	12 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Change in muscle strength as measured by handheld dynamometry	12 weeks
Change in five times sit-to-stand test	12 weeks
Change in 2-D and 3-D echocardiographic measurements	12 weeks
Change in accelerometry counts	12 weeks
Change in SWAY application balance assessments	12 weeks
Change in Patient Reported Outcomes	12 weeks
Change in Clinician Global Impression	12 weeks
Change in biomarkers	12 weeks
Number of participants with treatment related adverse events	12 weeks

Collaborators and Investigators

• Sponsor: Stealth BioTherapeutics Inc.

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2017
• Primary Completion (Actual): October 11, 2021
• Study Completion (Actual): December 2, 2021

Study Registration Dates

• First Submitted: March 21, 2017
• First Submitted That Met QC Criteria: March 27, 2017
• First Posted (Actual): April 4, 2017

Study Record Updates

• Last Update Posted (Actual): March 9, 2022
• Last Update Submitted That Met QC Criteria: March 7, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Keywords: Barth Syndrome; MTP-131; Stealth; elamipretide; Stealth BT; BTHS
• Additional Relevant MeSH Terms: Pathologic Processes; Heart Diseases; Cardiovascular Diseases; Metabolic Diseases; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Metabolism, Inborn Errors; Heart Defects, Congenital; Cardiovascular Abnormalities; Abnormalities, Multiple; Lipid Metabolism, Inborn Errors; Syndrome; Barth Syndrome
• Other Study ID Numbers: SPIBA-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No