Expanded Access for ATB200/AT2221 for the Treatment of IOPD
Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease
1 other identifier
expanded_access
N/A
3 countries
4
Brief Summary
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 27, 2020
CompletedFirst Posted
Study publicly available on registry
March 31, 2020
CompletedSeptember 10, 2025
September 1, 2025
March 27, 2020
September 9, 2025
Conditions
Keywords
Interventions
Eligibility Criteria
You may qualify if:
- Patient is male or female and 0 to \< 18 years old.
- Patient has a diagnosis of Pompe disease, based on documentation of GAA genotyping demonstrating variants associated with IOPD.
- Patient does not currently qualify for or is unable to enroll (eg, due to location, etc) in any commercial drug-sponsored ongoing clinical trial.
- Patient is currently being treated with an approved ERT and is declining (eg, worsening in motor function, respiratory function/parameters, cardiac function, etc) based on treating physician's clinical judgement.
- The patient's legal guardian or representative has given informed consent (and assent when age appropriate for regional requirements) to treatment prior to administering ATB200/AT2221 in a manner consistent with all national requirements.
- If of reproductive potential and sexually active, female subjects agree to use a highly effective method of contraception throughout the duration of the treatment and for up to 90 days after their last dose of ATB200/AT2221.
You may not qualify if:
- 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 and/or Amicus, pose an undue safety risk to the patient.
- 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
Study Sites (4)
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
UF Health Shands Hospital
Gainesville, Florida, 32610, United States
University Hospital of Padova
Padua, Italy
National Taiwan University Hospital
Taipei, 10002, Taiwan