Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional
Official Title: An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of neoGAA (GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidas
Brief Summary:
Primary Objective:
To evaluate the safety profile of neoGAA in patients with infantile-onset Pompe disease (IOPD) previously treated with alglucosidase alfa.
Secondary Objective:
To characterize the pharmacokinetic profile of neoGAA and to evaluate the preliminary efficacy of neoGAA in comparison to alglucosidase alfa.