Expanded Access for ATB200/AT2221 for the Treatment of IOPD

September 9, 2025 updated by: Amicus Therapeutics

Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease

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

Study Overview

Status

Available

Conditions

Intervention / Treatment

Detailed Description

This program is being offered on a patient by patient basis.

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Padua, Italy
        • Available
        • University Hospital of Padova
  • Taiwan
      • Taipei, Taiwan, 10002
        • Available
        • National Taiwan University Hospital
  • United States
    • California
      • Oakland, California, United States, 94609
        • Available
        • UCSF Benioff Children's Hospital Oakland
    • Florida
      • Gainesville, Florida, United States, 32610
        • Available
        • UF Health Shands Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second to 17 years (Child)

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

  1. Patient is male or female and 0 to < 18 years old.
  2. Patient has a diagnosis of Pompe disease, based on documentation of GAA genotyping demonstrating variants associated with IOPD.
  3. Patient does not currently qualify for or is unable to enroll (eg, due to location, etc) in any commercial drug-sponsored ongoing clinical trial.
  4. 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.
  5. 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.
  6. 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.

Exclusion Criteria:

  1. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221
  2. 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.
  3. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb,or Volibo)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Amicus Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

March 27, 2020

First Submitted That Met QC Criteria

March 27, 2020

First Posted (Actual)

March 31, 2020

Study Record Updates

Last Update Posted (Actual)

September 10, 2025

Last Update Submitted That Met QC Criteria

September 9, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ATB200-15

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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