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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03865836
Expanded Access Status : Available
First Posted : March 7, 2019
Last Update Posted : July 20, 2022
Information provided by (Responsible Party):
Amicus Therapeutics

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

Condition or disease Intervention/treatment
Pompe Disease Biological: ATB200 Drug: AT2221

Detailed Description:
This program is being offered on a patient by patient basis and will require company, IRB/IEC and single patient IND approval.

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Study Type : Expanded Access
Expanded Access Type : Individual Patients
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Named Patient or Compassionate Use for Treatment Use of ATB200/AT2221 for Patients With Pompe Disease

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03865836

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Contact: For Site 609-662-2000 PompeSiteInfo@amicusrx.com
Contact: For Patient 609-662-2000 patientadvocacy@amicusrx.com

Sponsors and Collaborators
Amicus Therapeutics
Additional Information:
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Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT03865836    
Other Study ID Numbers: ATB200-11
First Posted: March 7, 2019    Key Record Dates
Last Update Posted: July 20, 2022
Last Verified: July 2022
Keywords provided by Amicus Therapeutics:
expanded access
compassionate use
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases