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

• Status: Recruiting
• Trial end date: 2025-02-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amicus Therapeutics
• Treatments: Miglustat

Criteria

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 based on their current approved rhGAA dose and frequency (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)