Tests cipaglucosidase alfa with miglustat in children with infantile-onset Pompe disease
Tests cipaglucosidase alfa with miglustat in children under 18 with infantile-onset Pompe disease.
Plain-language title generated from the registry record
Registry title: A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Key facts
- Where
- Giessen, Heidelberg, Hochheim Am Main, and Münster, GermanySee the 4 sites
- Phase
- Phase 3
Where in Germany
- Justus Liebig University GiessenGiessen, GermanyTaking people
- Universitaetsklinikum Heidelberg AöRHeidelberg, GermanyTaking people
- SphinCS GmbHHochheim Am Main, GermanyTaking people
- Universitaetsklinikum Muenster AöRMünster, GermanyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Amicus Therapeutics
- Trial code
- NCT04808505EU 2022-501095-25-01
- Record updated
- 22 January 2026
- Original description
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 subjects with IOPD.Read full original descriptionShow less
An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, 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
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 subjects with IOPD.
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):; 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: 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; Cohort 1 and Cohort 2, unless specified; Subject requires invasive ventilation (eg, tracheostomy); Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1).