Phase 3
N=12
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
Barth Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT03098797 ↗Enrolled (actual)
12
Serious AEs
14.7%
Results posted
Apr 2024
Primary outcome: Primary: Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit — 400.1; 412.6; 443.1; 443.9 meters
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Elamipretide (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- Male
- Sponsor
- Stealth BioTherapeutics Inc.
- Primary completion
- Oct 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part 1: Distance Walked During the 6-Minute Walk Test (6MWT) by Visit |
400.1; 412.6; 443.1; 443.9 | — |
| PRIMARY Part 1: Total Fatigue Score Based on the BTHS-SA by Visit. |
7.672; 7.433; 6.837; 6.994; 6.220; 6.485 | — |
| SECONDARY Part 2: Distance Walked During the 6MWT |
45.4; 75.6; 79.8; 100.4; 68.0; 112.6 | — |
| SECONDARY Part 2: Change From Baseline: Total Fatigue Score (Q1, Q2, and Q4) Based on the BTHS-SA by Visit |
-2.425; -1.540; -1.800; -1.314; -1.500; -0.314 | — |
| SECONDARY Part 1: Muscle Strength as Measured by HHD by Visit |
131.236; 123.100; 135.934; 129.253 | — |
| SECONDARY Part 2: Change From Baseline: Muscle Strength by HHD (Newtons) by Visit |
33.606; 42.147; 54.073; 54.068; 68.798; 48.351 | — |
| SECONDARY Part 1: 5XSST by Visit |
12.965; 13.710; 14.023; 13.749 | — |
| SECONDARY Part 2: Change From Baseline: 5X Sit to Stand by Visit |
-0.773; 0.400; -1.505; 0.384; -2.533; -1.160 | — |
| SECONDARY Part 1: SWAY Application Balance Assessments by Visit |
70.752; 68.820; 78.698; 71.353 | — |
| SECONDARY Part 2: Change From Baseline: SWAY Application Balance Assessment |
-3.152; 10.464; 12.190; 6.664; 0.807; 9.484 | — |
| SECONDARY Part 1: Patient Global Impression Scales of Symptoms |
1.7; 1.8; 1.7; 1.5; 1.4; 1.6 | — |
| SECONDARY Part 2: Change From Baseline: Patient Global Impression Scales of Symptoms by Visit |
0.0; -0.4; -0.2; -0.6; -0.3; -0.4 | — |
| SECONDARY Part 1: Part 1: Clinician Global Impression |
1.8; 1.4; 1.6; 1.6 | — |
| SECONDARY Part 2: Change From Baseline: CGI Symptom Scale |
-0.4; 0.0; -1.3; -0.4; -1.3; -0.2 | — |
Summary
A randomized, double-blind cross over trial to evaluate the safety, efficacy, and tolerability of elamipretide in subjects with Barth syndrome.
Eligibility Criteria
Inclusion Criteria
- Genetically confirmed Barth Syndrome
- Male aged 12 and above
- At the screening visit, eGFR must meet the following:
- Body weight >30 kg AND eGFR ≥ 90mL/min at screening
- Body weight >40kg AND eGFR ≥60 but <90mL/min/ 1.73m² at screening
- Ambulatory and impaired during the baseline 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 occurrence 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
Data sourced from ClinicalTrials.gov (NCT03098797). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.