Summary

Eligibility
for people ages up to 17 years (full criteria)
Location
at Oakland, California and other locations
Dates
study started

Description

Summary

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

Official Title

Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease

Details

This program is being offered on a patient by patient basis.

Keywords

Pompe Disease Infantile-Onset expanded access compassionate use Glycogen Storage Disease Type II ATB200

Eligibility

You can join if…

Open to people ages up to 17 years

  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.

You CAN'T join if...

  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)

Locations

  • UCSF Benioff Children's Hospital Oakland not accepting new patients
    Oakland California 94609 United States
  • UF Health Shands Hospital not accepting new patients
    Gainesville Florida 32610 United States

Details

Status
not accepting new patients
Start Date
Sponsor
Amicus Therapeutics
ID
NCT04327973
Study Type
Expanded Access
Last Updated