Status:
RECRUITING
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Lead Sponsor:
Amicus Therapeutics
Conditions:
Glycogen Storage Disease Type II Infantile Onset
Eligibility:
All Genders
Up to 17 years
Phase:
PHASE3
Brief 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 sub...
Eligibility Criteria
Inclusion
- Cohort 1:
- Male or female subjects who are aged 6 months to \< 18 years on Day 1
- Subject must have documentation of IOPD genotype
- Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
- Subject must have received ERT for at least 6 months immediately before enrollment. For subjects whose ERT dosage has been modified, the subject must have been on the modified dosage and regimen for at least 3 months before enrollment
- Subjects aged ≥ 12 to \< 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; Subjects aged ≥ 5 to \< 12 years must perform one valid 6MWT (≥ 40 meters) at screening; Subjects aged 18 months to \< 5 years must be ambulatory and assessed to be likely to be able to perform 6MWT (≥ 40 meters) when they turn 5 years old
- Subjects must have experienced a clinical decline on their current rhGAA dose and frequency
- Cohort 2:
- Male or female subjects who are aged 0 to \<6 months at Day 1
- Subject must have documentation of IOPD genotype
- Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
- Subject is ERT-naïve
- Long-term Extension (Cohort 1 or Cohort 2):
- 1\. Subject must have, in the opinion of the investigator, benefited from therapy with cipaglucosidase alfa/miglustat during the 104-week primary treatment period with no significant safety concerns.
Exclusion
- Cohort 1 and Cohort 2, unless specified
- Subject requires invasive ventilation (eg, tracheostomy)
- Subject is CRIM negative and has not received prophylactic immunomodulation (Cohort 1); Subject is CRIM negative and will not be receiving prophylactic immunomodulation (Cohort 2)
- Subject has a history of life-threatening IARs/hypersensitivity (eg, anaphylaxis and severe cutaneous reactions) to ERT (eg, alglucosidase alfa, cipaglucosidase alfa, miglustat) or other iminosugars, or to any of the excipients, where rechallenge was unsuccessful
- Subject has prior history of illness or condition known to affect motor function
- Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)
Key Trial Info
Start Date :
July 18 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 1 2027
Estimated Enrollment :
36 Patients enrolled
Trial Details
Trial ID
NCT04808505
Start Date
July 18 2023
End Date
April 1 2027
Last Update
November 17 2025
Active Locations (14)
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1
University of Florida Clinical Research Center
Gainesville, Florida, United States, 32610
2
The Emory Clinic
Atlanta, Georgia, United States, 30322
3
Duke University Early Phase Research Unit
Durham, North Carolina, United States, 27710
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 54229