Study Details
A study in children with Late-Onset Pompe testing Cipaglucosidase Alfa/Miglustat.
(IRB#: IRB_00160440)
This study purpose is to learn if a new drug combination called cipaglucosidase alfa (ATB200) and miglustat (AT2221) can help children with Pompe disease. All children in this study will be treated with these drugs. The treatment will be given every two weeks. Cipaglucosidase alfa is given as an IV infusion. Miglustat is given as an oral pill. Treatment will last for about 52 weeks. Medical tests will be done during the study to track the health of participants.
- All genders
- Under 18 years old
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: All genders
Age: Under 18 years old
Volunteers: Volunteers with special conditions
Location: In Person
Will I be paid for my time?
Yes
IRB#: IRB_00160440
PI: David Viskochil
Department: PEDIATRIC GENETICS
Approval Date: 2023-08-09 06:00:00
Specialties: Pediatric Genetics