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
This trial does not accept healthy volunteersQualifiers
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
Biological/VaccineParticipants received ATB200 co-administered with AT2221 capsule (Miglustat)
AT2221
DrugParticipants received ATB200 co-administered with AT2221 capsule (Miglustat)
Treatment groups
No treatment groups listed
Trial outcomes
This trial has no outcomes listed.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov