Expanded Access for ATB200/AT2221 for the Treatment of IOPD
ConditionPompe Disease Infantile-Onset
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
Age0-17
SponsorAmicus Therapeutics
About this trial
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Eligibility criteria
Qualifiers
Patient is male or female and 0 to < 18 years old.
Patient has a diagnosis of Pompe disease, based on documentation of GAA genotyping demonstrating variants associated with IOPD.
Patient does not currently qualify for or is unable to enroll (eg, due to location, etc) in any commercial drug-sponsored ongoing clinical trial.
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.
Disqualifiers
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 and/or Amicus, pose an undue safety risk to the patient.
Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb,or Volibo)
Trial design
Treatments tested in this trial
- ATB200
- AT2221
Treatment groups
No treatment groups listed
Sponsors and collaborators
Source ClinicalTrials.gov