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

September 9, 2025 updated by: Amicus Therapeutics

Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With 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 and will require company, IRB/IEC and single patient IND approval.

Study Type

Expanded Access

Expanded Access Type

  • Individual Patients

Contacts and Locations

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

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

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

  1. Subject must have a diagnosis of Pompe disease based on documentation of one of the following:

    1. deficiency of GAA enzyme
    2. GAA genotyping
  2. Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)
  3. 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.

Exclusion Criteria:

  1. Patient, whether male or female, is planning to conceive a child during the treatment program.
  2. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221
  3. 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.
  4. Patient has received any gene therapy at any time.
  5. 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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

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 5, 2019

First Submitted That Met QC Criteria

March 5, 2019

First Posted (Actual)

March 7, 2019

Study Record Updates

Last Update Posted (Estimated)

September 15, 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

  • ATB200-11

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.

Clinical Trials on Pompe Disease

Clinical Trials on ATB200

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Pakistan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe