A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Recruiting now · Phase 3
Conditions studied: Glycogen Storage Disease Type II Infantile Onset
In brief
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.
Key facts
- Study ID
- NCT04808505
- Run by
- Amicus Therapeutics
- People needed
- 36
- Starts
- 2023-07-18
- Expected to finish
- 2027-07-01
- Last updated by the study team
- 2026-01-22
Who can join
Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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)
Where it is running
- Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom (enrolling)
- The Emory Clinic — Atlanta, Georgia, United States (enrolling)
- Duke University Early Phase Research Unit — Durham, North Carolina, United States (enrolling)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (enrolling)
- UPMC Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States (enrolling)
- University of Utah, Clinical and Translational Sciences Institute — Salt Lake City, Utah, United States (enrolling)
- Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie — Giessen, Germany (enrolling)
- Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS) — Heidelberg, Germany (enrolling)
- SphinCS GmbH — Höchheim, Germany (enrolling)
- Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1 — Münster, Germany (enrolling)
- AOU Federico II — Naples, Italy (enrolling)
- Erasmus MC, Sophia Kinderziekenhuis — Rotterdam, Netherlands (enrolling)
- University of Florida Clinical Research Center — Gainesville, Florida, United States (enrolling)
- National Taiwan University Hospital — Taipei, Taiwan
Full record on ClinicalTrials.gov
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