Artikel ; Online: Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER.
Genetics in medicine : official journal of the American College of Medical Genetics
2024 Band 26, Heft 7, Seite(n) 101138
Abstract: Purpose: Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS).: Methods: TAZPOWER was a 28-week randomized, double-blind, and placebo-controlled ... ...
Abstract | Purpose: Evaluate long-term efficacy and safety of elamipretide during the open-label extension (OLE) of the TAZPOWER trial in individuals with Barth syndrome (BTHS). Methods: 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. Results: 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. Conclusion: Elamipretide was associated with sustained long-term tolerability and efficacy, with improvements in functional assessments and cardiac function in BTHS. |
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Sprache | Englisch |
Erscheinungsdatum | 2024-04-08 |
Erscheinungsland | United States |
Dokumenttyp | Journal Article |
ZDB-ID | 1455352-1 |
ISSN | 1530-0366 ; 1098-3600 |
ISSN (online) | 1530-0366 |
ISSN | 1098-3600 |
DOI | 10.1016/j.gim.2024.101138 |
Datenquelle | MEDical Literature Analysis and Retrieval System OnLINE |
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