Phase 4 Rare Disease Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who are willing and able to give written consent before any trial procedures begin (for younger participants, a parent or legal guardian must also give written consent, and the participant may need to give their own agreement depending on local rules)
• People who are willing to follow all trial requirements for the full duration of the trial
• People who have been diagnosed with Barth Syndrome confirmed by genetic testing showing a change (pathogenic variant) in the TAZ gene
• Males aged 5 years or older at the time of the screening visit
• People whose heart is pumping at a normal or near-normal level (a left ventricular ejection fraction of 50% or higher), as measured by a specific type of heart ultrasound (3-D echocardiogram) at the screening visit
• People who have a history of heart muscle disease and have been on a stable, unchanged heart failure medication routine for at least 3 months before the screening visit
• People who are able to take the trial medication themselves, or who have a suitable family member or caregiver who can administer it for them
• Participants who have female partners who could become pregnant must be willing to use a highly effective form of contraception from the time of signing consent until 28 days after the last dose of trial medication

Who may not be able to join:

• People who are unable to complete the required physical tests or have a heart ultrasound (echocardiogram)
• People who have had a solid organ transplant, unless it was a successful heart transplant more than 12 months before screening, with no signs of rejection and stable post-transplant medications (confirm with trial site)
• People who have an implanted heart device (ICD) that has discharged in the 3 months before the screening visit
• People who are currently on a waiting list for a heart transplant
• People who have been hospitalised for heart failure within the 6 months before the screening visit
• People with any other illness or medical condition that, in the trial doctor's opinion, would prevent safe and reliable participation or could affect the trial results
• People who have a history of a systemic illness involving high levels of a type of white blood cell called eosinophils
• People whose kidneys are not functioning well enough, based on a specific blood test result at screening (confirm with trial site)
• People with an active cancer, or who have been cancer-free for less than 2 years — certain treated skin cancers may be an exception (confirm with trial site)
• People who have taken part in another clinical trial of an investigational drug or device within 30 days or 5 half-lives (whichever is longer) before screening, or who are in a non-interventional trial that might affect the results of this trial
• People who have had an allergic reaction to the trial medication or any of its ingredients
• People who have previously taken part in any trial or expanded access programme involving the drug elamipretide

Important: Always verify eligibility with the trial site directly before applying.