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Clinical Trial in Patients With Barth Syndrome- 4TAZPower

NCT07531251 · Status: NOT_YET_RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 48

Last updated 2026-05-06

No results posted yet for this study

Summary

Phase 3b/4, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy, safety, and pharmacokinetics of a once daily SC injection of elamipretide in subjects with genetically confirmed BTHS for 72 weeks. The primary trial objective is to confirm the efficacy of elamipretide which is approved in the United States(FORZINITY™) under the accelerated approval based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint.

Conditions

  • Barth Syndrome

Interventions

DRUG

Elamipretide

sub cutaneous injection

DRUG

Placebo

sub cutaneous injection

Sponsors & Collaborators

  • Stealth BioTherapeutics Inc.

    lead INDUSTRY

Principal Investigators

  • Rekha Sathyanarayana · Stealth BioTherapeutics Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
5 Years
Max Age
55 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-30
Primary Completion
2029-09-30
Completion
2029-11-30
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07531251 on ClinicalTrials.gov