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
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|Condition or disease||Intervention/treatment|
|Pompe Disease||Biological: ATB200 Drug: AT2221|
|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|
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.
|Ages Eligible for Study:||Child, Adult, Older Adult|
|Sexes Eligible for Study:||All|
Subject must have a diagnosis of Pompe disease based on documentation of one of the following:
- deficiency of GAA enzyme
- GAA genotyping
- Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)
- 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.
- Patient, whether male or female, is planning to conceive a child during the treatment program.
- Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221
- 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.
- Patient has received any gene therapy at any time.
- Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)
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
|Contact: For Site||609-662-2000||PompeSiteInfo@amicusrx.com|
|Contact: For Patientfirstname.lastname@example.org|
|Responsible Party:||Amicus Therapeutics|
|Other Study ID Numbers:||
|First Posted:||March 7, 2019 Key Record Dates|
|Last Update Posted:||July 20, 2022|
|Last Verified:||July 2022|
Glycogen Storage Disease Type II
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Glycogen Storage Disease
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases