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

• Status: Active, not recruiting
• Trial end date: 2021-12-01
• Target enrollment: 0
• Participant gender: Male

Summary

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

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Stealth BioTherapeutics Inc.

Criteria

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 at screening

• Ambulatory and impaired during the 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 occurrance 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