AVAILABLENot Applicable

Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease

Plain-language summary

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

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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.

✕. 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)

NCT IDNCT03865836

SponsorAmicus Therapeutics

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS

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