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

• Sponsor: Amicus Therapeutics (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04808505
• Collaborator: (none)
• Enrollment: 22
• Location: 1
• Arms: 3
• Anticipated Duration (Months): 30.1
• Patients Per Site Per Month: 0.7

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Glycogen Storage Disease Type II Infantile Onset	Biological: Cipaglucosidase alfa (ATB200); Drug: Miglustat (AT2221)	Phase 3

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 22 participants
• Allocation: Non-Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: 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
• Anticipated Study Start Date: Aug 1, 2022
• Anticipated Primary Completion Date: Feb 1, 2025
• Anticipated Study Completion Date: Feb 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cipaglucosidase Alfa/Miglustat -treated ERT-experienced subjects experiencing clinical decline	Biological: Cipaglucosidase alfa (ATB200); Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules; Drug: Miglustat (AT2221); Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules
Experimental: Cipaglucosidase Alfa/Miglustat treated ERT-naïve subjects	Biological: Cipaglucosidase alfa (ATB200); Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules; Drug: Miglustat (AT2221); Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsules
No Intervention: Approved rhGAA-treated ERT-naive subjects

Outcome Measures

Primary Outcome Measures

1. Incidence of treatment-emergent adverse events (TEAEs) from baseline [52 weeks]

2. Proportion of subjects with the composite endpoint at the end of Stage 1 for Cohort 2 and Cohort 3 [52 weeks]

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 17 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Florida Clinical Research Center	Gainesville	Florida	United States	32610

Sponsors and Collaborators

• Amicus Therapeutics

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Amicus Therapeutics
• ClinicalTrials.gov Identifier: NCT04808505
• Other Study ID Numbers: ATB200-08
• First Posted: Mar 22, 2021
• Last Update Posted: Jan 24, 2022
• Last Verified: Jan 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Glycogen Storage Disease Type II; Glycogen Storage Disease; Miglustat

Study Results

No Results Posted as of Jan 24, 2022