Trial Profile

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years

Status: Recruiting

Phase of Trial: Phase III

Latest Information Update: 12 Sep 2025

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At a glance

Drugs Cipaglucosidase alfa (Primary) ; Miglustat (Primary) ; Alglucosidase alfa
Indications Glycogen storage disease type II
Focus Adverse reactions; Registrational
Acronyms ROSSELLA
Sponsors Amicus Therapeutics

04 May 2023 Planned initiation date changed from 1 Apr 2023 to 1 Jun 2023.
18 Jan 2023 Planned End Date changed from 1 Feb 2025 to 1 Apr 2027.
18 Jan 2023 Planned primary completion date changed from 1 Feb 2025 to 1 Apr 2027.

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