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

An Open-label Study to Evaluate the Safety, Pharmacokinetics, Efficacy, 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


Lead Sponsor: Amicus Therapeutics

Source Amicus Therapeutics
Brief Summary

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-naïve pediatric subjects with classic IOPD.

Overall Status Not yet recruiting
Start Date 2021-08-01
Completion Date 2025-02-01
Primary Completion Date 2025-02-01
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Incidence of treatment-emergent adverse events (TEAEs) from baseline 52 weeks
Proportion of subjects with the composite endpoint at the end of Stage 1 for Cohort 2 and Cohort 3 52 weeks
Enrollment 22

Intervention Type: Biological

Intervention Name: Cipaglucosidase alfa (ATB200)

Description: Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules

Intervention Type: Drug

Intervention Name: Miglustat (AT2221)

Description: Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules



Inclusion Criteria: 1. Male or female subjects who are aged < 18 years at screening (For Cohort 1), 0 to <6 month (For Cohort 2 &3) 2. Confirmed diagnosis of classic IOPD 3. Subjects must have experienced a clinical decline on current standard of care (SOC) (For Cohort 1) Exclusion Criteria: 1. 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. 2. Subject requires invasive ventilation (eg, tracheostomy) or respiratory assistance as defined in study protocol 3. Subject has received any gene therapy at any time. 4. Subject who is CRIM-negative and has not received prophylactic immunomodulation (For Cohort 1) 5. Subject with high and sustained antibody titers as defined in study protocol (For Cohort 1) 6. Subject has any prior history of certain condition or any intercurrent illness as defined in study protocol 7. Subject has a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat 8. Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (For Cohort 1)



Minimum Age:


Maximum Age:

17 Years

Healthy Volunteers:


Overall Contact

Last Name: For Site

Phone: 609-662-2000

Email: [email protected]

Verification Date


Responsible Party

Type: Sponsor

Has Expanded Access Yes
Condition Browse
Number Of Arms 3
Arm Group

Label: Cipaglucosidase Alfa/Miglustat -treated ERT-experienced subjects experiencing clinical decline

Type: Experimental

Label: Cipaglucosidase Alfa/Miglustat treated ERT-naïve subjects

Type: Experimental

Label: Approved rhGAA-treated ERT-naive subjects

Type: No Intervention

Study Design Info

Allocation: Non-Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

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