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Expanded Access for ATB200/AT2221 for the Treatment of LOPD

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. Identifier: NCT03865836
Expanded Access Status : Available
First Posted : March 7, 2019
Last Update Posted : March 7, 2019
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
Official Title: Expanded Access for ATB200/AT2221 for the Treatment of Late Onset Pompe Disease Patients That do Not Qualify for Pivotal Study ATB200-03

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:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All

Inclusion Criteria:

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

    1. deficiency of GAA enzyme
    2. GAA genotyping
  2. Subject must provide signed informed consent prior to any study-related procedures being performed.

Exclusion Criteria:

  1. Subject is eligible for enrollment in an ongoing clinical trial using ATB200/AT2221.
  2. Subject, if female, is pregnant or breastfeeding at screening.
  3. Subject, whether male or female, is planning to conceive a child during the study.

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 identifier (NCT number): NCT03865836

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Contact: For Site 609-662-2000
Contact: For Patient 609-662-2000

Sponsors and Collaborators
Amicus Therapeutics

Additional Information:
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Responsible Party: Amicus Therapeutics Identifier: NCT03865836     History of Changes
Other Study ID Numbers: ATB200-11
First Posted: March 7, 2019    Key Record Dates
Last Update Posted: March 7, 2019
Last Verified: March 2019
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
Genetic Diseases, Inborn
Glycogen Storage Disease
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors