At a glance
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An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response
In Brief
A Phase 2 clinical trial evaluating Avalglucosidase alfa (GZ402666) and Alglucosidase alfa (GZ419829) for Glycogen Storage Disease Type II-Pompe's Disease. Active but no longer recruiting, targeting 22 participants across 12 sites in 5 countries.
Detailed Summary
This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.
Study Details
Timeline
Interventions
Pharmaceutical form: powder for concentrate for solution for infusion, Route of administration: IV
Pharmaceutical form: powder for concentrate for solution for infusion, Route of administration: IV