A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglu… (NCT04808505) | Clinical Trial Compass
RecruitingPhase 3
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
United States36 participantsStarted 2023-07-18
Plain-language 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 subjects with IOPD.
Who can participate
Age range17 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. 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
✓. Male or female subjects who are aged 0 to \<6 months at Day 1
✓. Subject must have documentation of IOPD genotype
✕. 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)
✕
What they're measuring
1
Proportion of subjects with infusion-associated reactions (IARs)
. 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)