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

  • End date
    Feb 3, 2025
  • participants needed
  • sponsor
    Amicus Therapeutics
Updated on 3 November 2021


This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-nave pediatric subjects with classic IOPD.

Condition Glycogen Storage Disease Type II Infantile Onset
Treatment Cipaglucosidase alfa (ATB200), Miglustat (AT2221)
Clinical Study IdentifierNCT04808505
SponsorAmicus Therapeutics
Last Modified on3 November 2021


Yes No Not Sure

Inclusion Criteria

Male or female subjects who are aged < 18 years at screening (For Cohort 1), 0 to <6 month (For Cohort 2 &3)
Confirmed diagnosis of classic IOPD
Subjects must have experienced a clinical decline based on their current approved rhGAA dose and frequency (For Cohort 1)

Exclusion Criteria

Subject received any investigational drug or any investigational biologic for Pompe disease within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before screening
Subject requires invasive ventilation (eg, tracheostomy) or respiratory assistance as defined in study protocol
Subject has received any gene therapy at any time
Subject who is CRIM-negative and has not received prophylactic immunomodulation (For Cohort 1)
Subject with high and sustained antibody titers as defined in study protocol (For Cohort 1)
Subject has any prior history of certain condition or any intercurrent illness as defined in study protocol
Subject has a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat
Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (For Cohort 1)
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