NCT03865836|Unknown
Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe Disease
1 other identifier
ATB200-11
Study Type
expanded_access
Target
N/A
Locations
0 countries
Sites
N/A
Timeline
RegisteredMar 2019
Brief Summary
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Trial Health
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 5, 2019
Completed2 days until next milestone
First Posted
Study publicly available on registry
March 7, 2019
CompletedLast Updated
September 15, 2025
Status Verified
September 1, 2025
First QC Date
March 5, 2019
Last Update Submit
September 9, 2025
Conditions
Keywords
expanded accesscompassionate use
Interventions
Eligibility Criteria
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
You may qualify if:
- Subject must have a diagnosis of Pompe disease based on documentation of one of the following:
- deficiency of GAA enzyme
- GAA genotyping
- Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)
- The patient must be willing to receive treatment with ATB200/AT2221 via this program, which includes signing an authorization form for sharing clinical data with Amicus Therapeutics, and its agent Early Access Care LLC.
You may not qualify if:
- Patient, whether male or female, is planning to conceive a child during the treatment program.
- Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221
- Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with or adversely impact protocol requirements.
- Patient has received any gene therapy at any time.
- Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Related Links
MeSH Terms
Conditions
Glycogen Storage Disease Type II
Condition Hierarchy (Ancestors)
Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases
Central Study Contacts
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 5, 2019
First Posted
March 7, 2019
Last Updated
September 15, 2025
Record last verified: 2025-09