1. Efficacy and Safety of ARRY-371797 in
- Author
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Calum A, MacRae, Matthew R G, Taylor, Luisa, Mestroni, John, Moses, Euan A, Ashley, Matthew T, Wheeler, Neal K, Lakdawala, Ray E, Hershberger, Victor, Sandor, Michael E, Saunders, Colleen, Oliver, Patrice A, Lee, and Daniel P, Judge
- Abstract
Lamin A/C gene (Patients withTwelve patients were enrolled; median (minimum, maximum) 6-minute walk test distance at baseline was 314 (246, 412) m. At week 12, the mean (80% CI) increase from baseline in 6-minute walk test distance was 69 (39, 100) m (median, 47 m). Median NT-proBNP concentration declined from 1409 pg/mL at baseline to 848 pg/mL at week 12. Mean left ventricular ejection fraction was stable at week 12. There was a trend toward improvement in Kansas City Cardiomyopathy Questionnaire Overall and Clinical Summary scores at week 12. No clinically significant drug-related safety concerns were identified.ARRY-371797 was well tolerated and resulted in increases in functional capacity and lower concentrations of cardiac biomarker NT-proBNP in patients withURL: https://clinicaltrials.gov; Unique identifier: NCT02057341.
- Published
- 2022