Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
ConditionPompe Disease
Trial statusAvailable
Trial phaseNot listed
Trial typeExpanded Access
Biological sexAll
AgeNot listed
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
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.
Disqualifiers
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.
Trial design
Treatments tested in this trial
- ATB200
- AT2221
Treatment groups
No treatment groups listed
Locations
Sponsors and collaborators
Source ClinicalTrials.gov