Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naive patients with late-onset Pompe disease: A pooled analysis of clinical trials.

ABSTRACT:

Background

The efficacy of avalglucosidase alfa (AVA) versus alglucosidase alfa (ALG) on forced vital capacity percent predicted (FVCpp) in patients with late-onset Pompe disease (LOPD) has been assessed in the Phase 3 COMET trial (NCT02782741). Due to the rarity of LOPD and thus small sample size in COMET, additional data were analyzed to gain further insights into the efficacy of AVA versus ALG.

Methods

Data from treatment-naive patients with LOPD were pooled from COMET and Phase 1/2 NEO1/NEO-EXT (NCT01898364/NCT02032524) trials for patients treated with AVA, and Phase 3 LOTS trial (NCT00158600) for patients treated with ALG. Regression analyses using mixed models with repeated measures consistent with those pre-specified in COMET were performed post-hoc. Analyses were adj

SUBMITTER: Mozaffar T 

PROVIDER: S-EPMC11259910 | biostudies-literature | 2024 Sep

REPOSITORIES: biostudies-literature