Barth Syndrome
9
1
1
4
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
0.0%
0 terminated out of 9 trials
100.0%
+13.5% vs benchmark
11%
1 trials in Phase 3/4
25%
1 of 4 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 4 completed trials
Clinical Trials (9)
Clinical Trial in Patients With Barth Syndrome- 4TAZPower
Global Registry and Natural History Study for Mitochondrial Disorders
Early Check: Expanded Screening in Newborns
An Intermediate Size Expanded Access Protocol of Elamipretide
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome
Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism
Heart and Muscle Metabolism in Barth Syndrome
Resistance Exercise in Barth Syndrome
Exercise Training in Barth Syndrome