Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
- Conditions
- Pompe Disease
- Registration Number
- NCT03865836
- Lead Sponsor
- Amicus Therapeutics
- Brief Summary
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/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.
Recruitment & Eligibility
- Status
- AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria
-
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.
Exclusion Criteria
- 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)
Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method