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

Amicus Therapeutics

Status

Conditions: Pompe Disease

Treatments: Biological: ATB200; Drug: AT2221

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT03865836

ATB200-11

Details and patient eligibility

About

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

Full description

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

Sex: All

Volunteers: No Healthy Volunteers

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)