Skip to content

Trial · Interventional

CompletedPhase 2/3Results postedRegistry data

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

Short name TAZPOWER

NCTNCT03098797 (opens ClinicalTrials.gov in a new tab)Other idSPIBA-201
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 2/3
Enrollment
12 participants (actual)
Sponsor
Stealth BioTherapeutics Inc. · Industry
Design
Randomized · Quadruple masking
Countries
1 country

United States

Start
5 Jul 2017
Primary completion
5 Oct 2018
Completion
11 Oct 2021
First posted
4 Apr 2017
Last update posted
16 Apr 2024
Conditions
Barth Syndrome

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
201720182019202020212022
  1. Ph 2/3

    TAZPOWER

    NCT03098797

    5 Jul 2017 to 11 Oct 2021

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

Interventions

  • ElamipretideDrug
  • PlaceboDrug

Arms2

  • Experimental

    Elamipretide, then Placebo

    Experimental Part 1: 12 weeks of single daily SC doses of 40 mg elamipretide in Treatment Period 1 followed by 12 weeks of treatment with placebo in Treatment Period 2 (separated by a 4 week washout period). Experimental Open Label Extension Part 2: All subjects will receive Elamipretide 40mg SC daily injections for up to 168 weeks

  • Placebo comparator

    Placebo , then Elamipretide

    Placebo Comparator Part 1: 12 weeks of single daily SC doses of placebo in Treatment Period 1 followed by 12 weeks of treatment with 40 mg elamipretide in Treatment Period 2 (separated by a 4 week washout period). Placebo Comparator Open Label Extension Part 2: All subjects will receive Elamipretide 40mg SC daily injections for up to 168 weeks

Primary outcomes2

Outcomes the sponsor listed as primary.

  1. Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit

    Time frame · Pre-dose to Week 12 (end of treatment)

    Registry description

    Average distance walked in meters during the 6-minute walk test (6MWT) at end of treatment, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.

  2. Part 1: Total Fatigue Score Based on the BTHS-SA by Visit.

    Time frame · Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment)

    Registry description

    Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit at predose, Weeks 1, 4, 8, and 12 (end of treatment). The BTHS-SA is 3-question fatigue assessment using a 0 to 4-point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects; a lower score means better outcome, higher score means a worse outcome. Combined Min score=0, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Results from each 12-week period were combined, where end of treatment for Period 1 =Visit 5 (week 12), and End of Period 2 = Visit 10 (week 12)

Secondary outcomes(12)

  1. Part 2: Distance Walked During the 6MWT

    Time frame · Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192

    Registry description

    Change from baseline for distance walked in meters during the 6-minute walk test (6MWT) by visit where Baseline= Part 1 Period 1 Pre-dose

  2. Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit

    Time frame · Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192

    Registry description

    Change from Baseline (mean) in Total Fatigue Score (Q1, Q2, and Q4) Based on the BarTH Syndrome Symptom Assessment (BTHS-SA) by visit. The BTHS-SA is 3-question fatigue assessment using a 0-4 point scale for each question where no fatigue =0 and very severe fatigue =4, with scores from all 3 questions combined and averaged for all subjects by visit; a lower score means better outcome, higher score means a worse outcome. Change from baseline: an increase in scores would mean worse outcome, a decrease in scores means better outcome. Combined Min score=-12, max score =12. Weekly values are based on the weekly average of the last 7 days before the site or visiting nurse visit for both periods. Baseline= Period 1 Predose.

  3. Part 1: Muscle Strength as Measured by HHD by Visit

    Time frame · Pre-dose to Week 12 (end of treatment)

    Registry description

    Muscle strength as measured by handheld dynamometry (HHD) in Newtons, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. Handheld dynamometry assesses the average strength of the knee extensors of both legs.

  4. Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit

    Time frame · Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192

    Registry description

    Muscle strength as measured by handheld dynamometry (HHD) in Newtons. Handheld dynamometry assesses the average strength of the knee extensors of both legs. Change from baseline where Baseline= Part 1 Period 1 Pre-dose: the greater positive change, the better the outcome, the smaller positive change (or negative change) the number the worse the outcome.

  5. Part 1: 5XSST by Visit

    Time frame · Pre-dose to Week 12 (end of treatment)

    Registry description

    Time (in seconds) to complete the Five Times Sit-To-Stand, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.

  6. Part 2: Change From Baseline: 5X Sit to Stand by Visit

    Time frame · Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192

    Registry description

    Change in baseline in time (in seconds), where Baseline= Part 1 Period 1 Pre-dose to complete the Five Times Sit-To-Stand by visit. More time means worse outcome, less time means better outcome.

  7. Part 1: SWAY Application Balance Assessments by Visit

    Time frame · Pre-dose to Week 12 (end of treatment)

    Registry description

    SWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability from Pre-dose to Week 12 (end of treatment), where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.

  8. Part 2: Change From Baseline: SWAY Application Balance Assessment

    Time frame · Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192

    Registry description

    Change from Baseline: SWAY Application Balance Assessment,where Baseline= Part 1 Period 1 Pre-doseSWAY application balance assessment consists of five stances each performed for 10 seconds and scores postural stability on a scale from 0-100 with higher scores indicating better postural stability. An increase from baseline is a better outcome and a decrease in score is a worse outcome.

  9. Part 1: Patient Global Impression Scales of Symptoms

    Time frame · Pre-dose, Week 1, Week 12 (end of treatment)

    Registry description

    Patient-reported health status over the past week, by visit, where end of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period. Scores range from 0-40 for adolescents and 0-50 for adults. PGI Scale is as follows: 0=None, 1=Mild, 2=Moderate, 3=Severe, and 4=Very Severe. Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome.

  10. Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit

    Time frame · Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192

    Registry description

    Part 2: Change from Baseline: Patient Global Impression Scales of Symptoms by Visit Patient-reported health status over the past week where Baseline= Part 1 Period 1 Pre-dose, administered at Week 12 for Period 1 and Period 2. Scores range from 0-40 for adolescents and 0-50 for adults. PGI Scale is as follows: 1=None, 2=Mild, 3=Moderate, 4=Severe, and 5=Very Severe. Change from baseline: Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome.

  11. Part 1: Part 1: Clinician Global Impression

    Time frame · Pre-dose to Week 12 (end of treatment)

    Registry description

    Clinician Global Impression Scale by visit: Clinician-reported overall health status at end of treatment, administered at Week 12 for Period 1 and Period 2. Scores range from 0-6 for both adolescents and adults. PGI Scale is as follows: 1= Very much Better, 2= Moderately Better, 3= A Little Better, 4= No Change, and 5= A Little Worse 6= Very much Worse. Higher score means worse health status, means worse outcome; lower score means better health status, means better outcome. End of treatment means end of Visit 5 (Week 12) for Period 1 and end of Visit 10 (Week 12) for Period 2. Results were pooled for each 12 week period.

  12. Part 2: Change From Baseline: CGI Symptom Scale

    Time frame · Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192

    Registry description

    Change from Baseline: CGI Symptom Scale by visit where Baseline= Part 1 Period 1 Pre-dose. Clinician Global Impression Scale: Clinician-reported overall health status at end of treatment, administered at Week 12 for Period 1 and Period 2. Scores range from 0-6 for both adolescents and adults. PGI Scale is as follows: 1= Very much Better, 2= Moderately Better, 3= A Little Better, 4= No Change, and 5= A Little Worse 6= Very much Worse. Change from baseline: higher score means worse health status, means worse outcome; lower score means better health status, means better outcome.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· Pre-dose to Week 12 (end of treatment)

Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit

GroupValue (meters)Spread
Experimental: Elamipretide400.155.07
Placebo412.660.16
Primary outcomeMean· Pre-dose, Weeks 1, 4, 8, and 12 (end of treatment)

Part 1: Total Fatigue Score Based on the BTHS-SA by Visit.

GroupValue (score on a scale)Spread
Experimental: Elamipretide7.6721.9360
Placebo7.4331.3848
Secondary and other outcome results(6)
Secondary outcomeMean· Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192

Part 2: Distance Walked During the 6MWT

GroupValue (meters)Spread
Part 1: Elamipretide (Sequence AB)45.455.73
Part 2: Placebo (Sequence BA)75.687.25
Secondary outcomeMean· Baseline to Weeks 12, 24, 36, 48, 72, 96, 120, 144, 168, 192

Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit

GroupValue (score on a scale)Spread
Part 2: OLE: (Treatment AB)-2.4252.0849
Part 2: OLE: (Treatment BA)-1.5401.3653
Secondary outcomeMean· Pre-dose to Week 12 (end of treatment)

Part 1: Muscle Strength as Measured by HHD by Visit

GroupValue (Newtons)Spread
Experimental: Elamipretide131.23629.9004
Placebo123.10024.6762
Secondary outcomeMean· Baseline to Weeks 12, 24, 36, 48, 72, 96,168, 192

Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit

GroupValue (Newtons)Spread
Part 2: OLE: (Treatment AB)33.60624.7304
Part 2: OLE: (Treatment BA)42.14734.9498
Secondary outcomeMean· Pre-dose to Week 12 (end of treatment)

Part 1: 5XSST by Visit

GroupValue (seconds)Spread
Experimental: Elamipretide12.9653.3366
Placebo13.7105.3088
Secondary outcomeMean· Baseline to Weeks 12, 24, 36, 48, 72, 96, 168, 192

Part 2: Change From Baseline: 5X Sit to Stand by Visit

GroupValue (seconds)Spread
Part 2: OLE: (Sequence AB)-0.7732.5323
Part 2: OLE: (Sequence BA)0.4002.7824

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Part 2 (OLE): Elamipretide (Sequence AB)Part 2: Participants from Part 1 who received 12 weeks of elamipretide first, placebo for the last 12 weeks of treatment, then proceeded to Part 2 OLE and received up to 192 weeks of daily subcutaneous injections of 40mg elamipretide.0 / 51 / 55 / 5
Part 2 (OLE): Elamipretide (Sequence BA)Part 2: Participants from Part 1 who received 12 weeks of placebo first, then elamipretide for the last 12 weeks of treatment, then proceeded to Part 2 OLE and received up to 192 weeks of daily subcutaneous injections of 40mg elamipretide.0 / 54 / 55 / 5
Part 1: ElamipretideParticipants who received Elamipretide either in the first or last 12 weeks of treatment.0 / 60 / 1212 / 12
Part 1: PlaceboParticipants who received Placebo either in the first or last 12 weeks of treatment.0 / 60 / 1212 / 12

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies3

Studies that cite this trial.

Studies with results

1 study on this page

Indexing hint: Randomized controlled trial · 2025

Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study.

Gwaltney et al. 2025Orphanet journal of rare diseasescited by 2

PMID 40281531PMC12032656DOI 10.1186/s13023-025-03693-5PUB-40281531

CC BY
Download PDF

Indexing hint: Randomized controlled trial · 2024

8 results

Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER.

Thompson et al. 2024Genetics in medicine : official journal of the American College of Medical Geneticscited by 29

PMID 38602181DOI 10.1016/j.gim.2024.101138PUB-38602181

Full text at the publisher8 results
Publisher pageSee results (8)

PubMed tag: none · 2022

Quality of life in Barth syndrome.

Kim et al. 2022Therapeutic advances in rare diseasecited by 2

PMID 37180415PMC10032447DOI 10.1177/26330040221093743PUB-37180415

Free to read · publisher termsCC BY-NC
Read at source

Eligibility

Who could join, as posted.

Sex
Male
Age
from 12 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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/ 1.73m² at screening * Ambulatory and impaired during the baseline 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 occurrence 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

PepBank is a research reference. Talk to a doctor before you use any peptide.