A Phase 3 interventional study of Cipaglucosidase alfa and Miglustat in Glycogen Storage Disease Type II Infantile Onset, sponsored by Amicus Therapeutics. Recruiting at 14 sites in 6 countries. Open to participants aged Up to 17 Years. Per ClinicalTrials.gov, last updated 2026-01-22.
Sponsored by Amicus Therapeutics (part of BioMarin Pharmaceutical) · Phase 3, Interventional, and Treatment
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Among 16 studies in Glycogen Storage Disease Type II by BioMarin Pharmaceutical — 2 open to participants now, 3 under way, 2 of them phase 3. Glycogen Storage Disease Type II · BioMarin Pharmaceutical.
Among the 16 studies in Glycogen Storage Disease Type II by BioMarin Pharmaceutical, this one is the 3rd largest by planned enrollment of the 10 interventional ones that give a count, is the only one that started in 2023, is one of 5 in phase 3, and was the 15th registered.
162 studies on the registry are indexed under Glycogen Storage Disease Type II; 34 are open to participants now.
Browse Glycogen Storage Disease Type II studies →2 of the 16 studies in Glycogen Storage Disease Type II by BioMarin Pharmaceutical are open now, in 12 countries. Recruiting first, the newest first:
Amicus Therapeutics · Sites in United States, Austria, Belgium and 9 more countries
By country, each count listing its studies:
Of the 23 Glycogen Storage Disease Type II studies recruiting now, 16 (70%) set no upper age limit and 6 (26%) stop at 75 or younger; 15 take children under 18; none take healthy volunteers.
This study: Up to 17 Years · all sexes · no healthy volunteers.
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16 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 2 · Phase 2 5 · Phase 3 5
First in 2007; this study’s year is marked. 1 started in 2023.
This study's planned enrollment of 36 is above the median of 20 across 89 interventional studies indexed under Glycogen Storage Disease Type II.
Median enrollment 25 across the 10 interventional studies, 21 across the 4 observational. Of the 11 that completed or were terminated, 9 (82%) have results posted.
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This study is registered under Amicus Therapeutics, one of the names counted as BioMarin Pharmaceutical. Under that name alone:
Amicus Therapeutics is the lead sponsor of 42 studies on the registry; 8 are open to participants now.
2 of the studies in Glycogen Storage Disease Type II by BioMarin Pharmaceutical that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT03911505Phase 3Amicus TherapeuticsZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD | 2027-03 |
| NCT04808505Phase 3Amicus TherapeuticsA Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 | 2027-07 |
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Among the 16 studies in Glycogen Storage Disease Type II by BioMarin Pharmaceutical, from their registry updates:
Nothing recorded among them since 2026-09-25.
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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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Cohort 1:
Cohort 2:
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 Criteria:
Cohort 1 and Cohort 2, unless specified
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Pediatric IOPD subjects 6 months to \<18 years experiencing clinical decline
Biological: Cipaglucosidase alfa · Drug: Miglustat
Pediatric IOPD subjects \<6 months
Biological: Cipaglucosidase alfa · Drug: Miglustat
Sterile lyophilized powder intravenous (IV) infusion
Also known as: ATB200
65 mg oral capsules
Also known as: AT2221
Proportion of subjects with infusion-associated reactions (IARs)
Time frame: 104 weeks
Not enrolling at this site.
Plan to share: No
No publications or documents are linked to this record.
Started Jul 2023 (actual). Primary completion is estimated for Jul 2027, 48 months after the start.
How these figures are worked out, and which studies are left out of them.
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Studies of the drugs tested here: Cipaglucosidase alfa · Miglustat
Glycogen Storage Disease Type II →