Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report
Journal
Genetics in medicine : official journal of the American College of Medical Genetics
Date Issued
2022-12-20
Author(s)
Kishnani, Priya S
Kronn, David
Brassier, Anaïs
Broomfield, Alexander
Davison, James
Hahn, Si Houn
Kumada, Satoko
Labarthe, François
Ohki, Hirotaka
Pichard, Samia
Prakalapakorn, S Grace
Haack, Kristina An
Kittner, Barbara
Meng, Xianzhang
Sparks, Susan
Wilson, Catherine
Zaher, Atef
Abstract
Mini-COMET (NCT03019406; Sanofi) is a phase 2, open-label, ascending-dose, 3-cohort study, evaluating avalglucosidase alfa safety, pharmacokinetics, and efficacy in individuals with infantile-onset Pompe disease aged <18 years who previously received alglucosidase alfa and showed clinical decline (cohorts 1 and 2) or suboptimal response (cohort 3).
Subjects
Acid α-glucosidase (GAA) deficiency; Alglucosidase alfa; Avalglucosidase alfa; Enzyme replacement therapy; Infantile-onset Pompe disease
SDGs
Type
journal article