Expanded Access for ATB200/AT2221 for the Treatment of IOPD

Available (expanded access)

Conditions studied: Pompe Disease Infantile-Onset

In brief

This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.

Key facts

Study ID

NCT04327973

Run by

Amicus Therapeutics

Last updated by the study team

2025-09-10

Who can join

Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.

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)

Where it is running

• UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
• UF Health Shands Hospital — Gainesville, Florida, United States
• University Hospital of Padova — Padova, Italy
• National Taiwan University Hospital — Taipei, Taiwan

Full record on ClinicalTrials.gov

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