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NCT07531251 · ClinicalTrials.gov registry record · Phase 4

Clinical Trial in Patients With Barth Syndrome- 4TAZPower

A Phase 4 study, sponsored by Stealth BioTherapeutics.

Not yet
Registry status
Phase 4
Development phase
48
Enrollment target

NCT07531251: Clinical Trial Phase 4 study, sponsored by Stealth BioTherapeutics.

NCT07531251 is a Phase 4 study that has not yet begun recruiting, run by Stealth BioTherapeutics. The registered enrollment target is 48 participants, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (91% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT07531251, a Phase 4 study, has not yet begun recruiting, sponsored by Stealth BioTherapeutics.

NOT YET RECRUITING
Registry status
Phase 4
Development phase
48 participants
Enrollment target

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.

Primary Outcome

-change in the composite normalized score of the three functional tests: Six-minute walk test (6MWT), the triple-timed up and go test (3TUG), and the Five times sit-to-stand test (5XSST) from baseline to 72 weeks of treatment

Interventions

  • DRUG Placebo
  • DRUG Elamipretide

Trial Details

FieldValue
Enrollment Target 48 participants
Start Date 2026-06-30
Est. Completion 2029-11-30
Phase Phase 4
Stealth BioTherapeutics

23 total trials

What is known before NCT07531251 opens enrollment

NCT07531251 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 48 participants, a relatively small participant target, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (91% lower).

The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.

NCT07531251 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT07531251 about?

NCT07531251 is a clinical study titled "Clinical Trial in Patients With Barth Syndrome- 4TAZPower". 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...

What is the current status of trial NCT07531251?

This trial is currently not yet recruiting. It is a Phase 4 study. The enrollment target is 48 participants. The study started on 2026-06-30. Estimated completion is 2029-11-30.

What interventions are being tested in trial NCT07531251?

The interventions under investigation include: Placebo (DRUG), Elamipretide (DRUG).

Who is sponsoring clinical trial NCT07531251?

This trial is sponsored by Stealth BioTherapeutics, which has 23 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT07531251's enrollment target sits among peer trials

48 1331st of 2000 higher than 653 of 2,000 other Phase 4 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 4 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT07531251, the US trial registry maintained by the National Library of Medicine. NCT07531251 (small enrollment · none site footprint · not yet recruiting) retrieved and formatted by PlainTrial, see methodology.