Clinical Trial

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Study acronym: ROSSELLA
Recruiting Phase 3
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Summary
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
Protocol Amendment History 22 amendments
This ClinicalTrials.gov record has been amended 22 times since 2021-03-17; most recent amendment 2026-01-20.
Status change: Not Yet Recruiting → Recruiting 2021-08-06
Trial Details
NCT Number NCT04808505
Lead Sponsor Amicus Therapeutics
Conditions Glycogen Storage Disease Type II Infantile Onset
Enrollment 36 participants
Start Date 2023-07-18
Primary Completion 2027-07 (estimated)
Study Completion 2027-07 (estimated)
Updated on ClinicalTrials.gov 2026-01-22