Study summary · research use only
Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Plain-language summary
Paraphrased from the published abstract below — not a verdict on whether anything works.
This human study reports 168-week open-label extension (OLE) results from the TAZPOWER trial evaluating elamipretide in patients with Barth syndrome (BTHS), following an initial 28-week randomized, double-blind, placebo-controlled phase. Patients in the OLE continued subcutaneous elamipretide 40 mg daily. Ten patients entered the OLE and 8 reached the week 168 visit; the most common adverse events were injection-site reactions. The abstract reports improvements from OLE baseline on the 6-minute walk test (6MWT) at all OLE time points, with a cumulative improvement of 96.1 m by week 168 (P = .003), along with improved BTHS Symptom Assessment Total Fatigue scores, improved three-dimensional left ventricular stroke, end-diastolic, and end-systolic volumes, and improved MLCL/CL biomarker values correlating with clinical outcomes.
Abstract
Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS). TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled trial followed by a 168-week OLE. Patients entering the OLE continued elamipretide 40 mg subcutaneous daily. OLE primary endpoints were safety and tolerability; secondary endpoints included change from baseline in the 6-minute walk test (6MWT) and BarTH Syndrome Symptom Assessment (BTHS-SA) Total Fatigue score. Muscle strength, physician- and patient-assessed outcomes, echocardiographic parameters, and biomarkers, including cardiolipin (CL) and monolysocardiolipin (MLCL), were assessed. Ten patients entered the OLE; 8 reached the week 168 visit. Elamipretide was well tolerated, with injection-site reactions being the most common adverse events. Significant improvements from OLE baseline on 6MWT occurred at all OLE time points (cumulative 96.1 m of improvement [week 168, P = .003]). Mean BTHS-SA Total Fatigue scores were below baseline (improved) at all OLE time points. Three-dimensional (3D) left ventricular stroke, end-diastolic, and end-systolic volumes improved, showing significant trends for improvement from baseline to week 168. MLCL/CL values showed improvement, correlating to important clinical outcomes. Elamipretide was associated with sustained long-term tolerability and efficacy, with improvements in functional assessments and cardiac function in BTHS.
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