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

Thompson WR, Manuel R, Abbruscato A, Carr J, Campbell J, Hornby B, Vaz FM, Vernon HJ

Genetics in medicine : official journal of the American College of Medical Genetics8 Apr 2024Cited by 29 (Europe PMC)

PMID38602181DOI10.1016/j.gim.2024.101138PepBankPUB-38602181

Publisher page PubMedAsk about this study

Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: CC BY-NC-ND. Results still quote short attributed sentences.

Results from this study8

8 results from studies that gave the peptide. 8 results from people.

01HumanNon-randomized trialSafety outcome
In patients with Barth syndrome, elamipretide was well tolerated during the 168-week open-label extension.
In plain wordsIn people with Barth syndrome, elamipretide was well tolerated during long-term open-label treatment.
Peptide
Elamipretide (SS-31)
Outcome
Not applicableTolerability
Study
n = 10
Analysis
Primary
Outcome group
Adverse events · Whole body
Studied dose
40 mg daily · subcutaneous · 168 weeksAs given in this study (human).
Source sentences
Summary
Elamipretide was well tolerated, with injection-site reactions being the most common adverse events.
3 context sentences(design, population, supporting numbers)
Summary · context
TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE.
Summary · context
Patients entering the OLE continued elamipretide 40 mg subcutaneous daily.
Summary · context
Ten patients entered the OLE; 8 reached the week 168 visit.
Report a problemFND-3860218101
02HumanNon-randomized trialSafety outcome
In patients with Barth syndrome, injection-site reactions were the most common adverse events during the open-label extension.
In plain wordsIn people with Barth syndrome, reactions at the injection site were the most common side effects of elamipretide.
Peptide
Elamipretide (SS-31)
Outcome
Not applicableInjection-site reactions
Study
n = 10
Analysis
Primary
Outcome group
Adverse events · Whole body
Studied dose
40 mg daily · subcutaneous · 168 weeksAs given in this study (human).
Source sentences
Summary
injection-site reactions being the most common adverse events.
3 context sentences(design, population, supporting numbers)
Summary · context
TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE.
Summary · context
Patients entering the OLE continued elamipretide 40 mg subcutaneous daily.
Summary · context
Ten patients entered the OLE; 8 reached the week 168 visit.
Report a problemFND-3860218102
03HumanNon-randomized trialEfficacy outcome
In patients with Barth syndrome, 6-minute walk distance improved significantly from OLE baseline at all time points, with a cumulative gain at week 168.
In plain wordsIn people with Barth syndrome, walking distance in six minutes improved from the start of long-term treatment with elamipretide.
Peptide
Elamipretide (SS-31)
Outcome
Increase6-minute walk test change from OLE baseline
Study
n = 8
Comparator
OLE baseline
Analysis
Secondary
Outcome group
Exercise and physical function · Musculoskeletal
Studied dose
40 mg daily · subcutaneous · 168 weeksAs given in this study (human).
Measurements
6MWT cumulative improvement
96.1 m
week 168
Source sentences
Summary
Significant improvements from OLE baseline on 6MWT occurred at all OLE time points (cumulative 96.1 m of improvement [week 168, P = .003]).
3 context sentences(design, population, supporting numbers)
Summary · context
TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE.
Summary · context
Patients entering the OLE continued elamipretide 40 mg subcutaneous daily.
Summary · context
Ten patients entered the OLE; 8 reached the week 168 visit.
Report a problemFND-3860218103
04HumanNon-randomized trialEfficacy outcome
In patients with Barth syndrome, mean BTHS-SA Total Fatigue scores were below baseline (improved) at all OLE time points.
In plain wordsIn people with Barth syndrome, reported fatigue scores improved from the start of long-term treatment with elamipretide.
Peptide
Elamipretide (SS-31)
Outcome
DecreaseBTHS-SA Total Fatigue score
Study
n: not reported in source
Comparator
OLE baseline
Analysis
Secondary
Outcome group
Exercise and physical function · Whole body
Studied dose
40 mg daily · subcutaneous · 168 weeksAs given in this study (human).
Source sentences
Summary
Mean BTHS-SA Total Fatigue scores were below baseline (improved) at all OLE time points.
3 context sentences(design, population, supporting numbers)
Summary · context
TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE.
Summary · context
Patients entering the OLE continued elamipretide 40 mg subcutaneous daily.
Summary · context
Ten patients entered the OLE; 8 reached the week 168 visit.
Report a problemFND-3860218104
05HumanNon-randomized trialEfficacy outcome
In patients with Barth syndrome, 3D left ventricular stroke volume improved, with a significant trend from baseline to week 168.
In plain wordsIn people with Barth syndrome, a heart pumping measure improved over long-term elamipretide treatment.
Peptide
Elamipretide (SS-31)
Outcome
Direction not reported3D left ventricular stroke volume
Study
n: not reported in source
Comparator
OLE baseline
Outcome group
Heart function · Cardiovascular
Studied dose
40 mg daily · subcutaneous · 168 weeksAs given in this study (human).
Source sentences
Summary
Three-dimensional (3D) left ventricular stroke, end-diastolic, and end-systolic volumes improved, showing significant trends for improvement from baseline to week 168.
3 context sentences(design, population, supporting numbers)
Summary · context
TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE.
Summary · context
Patients entering the OLE continued elamipretide 40 mg subcutaneous daily.
Summary · context
Ten patients entered the OLE; 8 reached the week 168 visit.
Report a problemFND-3860218105
06HumanNon-randomized trialEfficacy outcome
In patients with Barth syndrome, 3D left ventricular end-diastolic volume improved, with a significant trend from baseline to week 168.
In plain wordsIn people with Barth syndrome, a heart size measure improved over long-term elamipretide treatment.
Peptide
Elamipretide (SS-31)
Outcome
Direction not reported3D left ventricular end-diastolic volume
Study
n: not reported in source
Comparator
OLE baseline
Outcome group
Heart function · Cardiovascular
Studied dose
40 mg daily · subcutaneous · 168 weeksAs given in this study (human).
Source sentences
Summary
Three-dimensional (3D) left ventricular stroke, end-diastolic, and end-systolic volumes improved, showing significant trends for improvement from baseline to week 168.
3 context sentences(design, population, supporting numbers)
Summary · context
TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE.
Summary · context
Patients entering the OLE continued elamipretide 40 mg subcutaneous daily.
Summary · context
Ten patients entered the OLE; 8 reached the week 168 visit.
Report a problemFND-3860218106
07HumanNon-randomized trialEfficacy outcome
In patients with Barth syndrome, 3D left ventricular end-systolic volume improved, with a significant trend from baseline to week 168.
In plain wordsIn people with Barth syndrome, a heart size measure improved over long-term elamipretide treatment.
Peptide
Elamipretide (SS-31)
Outcome
Direction not reported3D left ventricular end-systolic volume
Study
n: not reported in source
Comparator
OLE baseline
Outcome group
Heart function · Cardiovascular
Studied dose
40 mg daily · subcutaneous · 168 weeksAs given in this study (human).
Source sentences
Summary
Three-dimensional (3D) left ventricular stroke, end-diastolic, and end-systolic volumes improved, showing significant trends for improvement from baseline to week 168.
3 context sentences(design, population, supporting numbers)
Summary · context
TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE.
Summary · context
Patients entering the OLE continued elamipretide 40 mg subcutaneous daily.
Summary · context
Ten patients entered the OLE; 8 reached the week 168 visit.
Report a problemFND-3860218107
08HumanNon-randomized trialEfficacy outcome
In patients with Barth syndrome, MLCL/CL values improved and correlated with important clinical outcomes.
In plain wordsIn people with Barth syndrome, a blood marker of the disease improved and was linked to clinical outcomes.
Peptide
Elamipretide (SS-31)
Outcome
Direction not reportedMLCL/CL ratio
Study
n: not reported in source
Comparator
OLE baseline
Outcome group
Mitochondrial function · Whole body
Studied dose
40 mg daily · subcutaneous · 168 weeksAs given in this study (human).
Source sentences
Summary
MLCL/CL values showed improvement, correlating to important clinical outcomes.
3 context sentences(design, population, supporting numbers)
Summary · context
TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE.
Summary · context
Patients entering the OLE continued elamipretide 40 mg subcutaneous daily.
Summary · context
Ten patients entered the OLE; 8 reached the week 168 visit.
Report a problemFND-3860218108

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 completed · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 2/3Results postedNCT03098797

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

Stealth BioTherapeutics Inc.n = 125 Jul 2017 to 11 Oct 2021

Barth Syndrome

Trial page ClinicalTrials.govUpdated 16 Apr 2024

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
9 Oct 2026, 18:17 UTC
Study results updated
Version 1
9 Oct 2026, 02:47 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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