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Expanded Access for ATB200/AT2221 for the Treatment of IOPD

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: NCT04327973
Expanded Access Status : Available
First Posted : March 31, 2020
Last Update Posted : September 9, 2020
Sponsor:
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 Infantile-Onset Biological: ATB200 Drug: AT2221

Detailed Description:
This program is being offered on a patient by patient basis.

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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
Official Title: Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease



Intervention Details:
  • Biological: ATB200
    Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
  • Drug: AT2221
    Participants received ATB200 co-administered with AT2221 capsule (Miglustat)

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Criteria

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)

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): NCT04327973


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

Locations
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United States, Florida
UF Health Shands Hospital Available
Gainesville, Florida, United States, 32610
Taiwan
National Taiwan University Hospital Available
Taipei, Taiwan, 10002
Sponsors and Collaborators
Amicus Therapeutics
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Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT04327973    
Other Study ID Numbers: ATB200-15
First Posted: March 31, 2020    Key Record Dates
Last Update Posted: September 9, 2020
Last Verified: September 2020
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