Trials / Available
AvailableNCT04327973
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
Expanded Use Of Recombinant Human Acid Alpha-Glucosidase/N-butyl-deoxynojirimycin (ATB200/AT2221) For Patients With Infantile-Onset Pompe Disease
- Status
- Available
- Phase
- —
- Study type
- Expanded Access
- Enrollment
- —
- Sponsor
- Amicus Therapeutics · Industry
- Sex
- All
- Age
- 0 Years – 17 Years
- Healthy volunteers
- —
Summary
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
Detailed description
This program is being offered on a patient by patient basis.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | ATB200 | Participants received ATB200 co-administered with AT2221 capsule (Miglustat) |
| DRUG | AT2221 | Participants received ATB200 co-administered with AT2221 capsule (Miglustat) |
Timeline
- First posted
- 2020-03-31
- Last updated
- 2025-09-10
Locations
4 sites across 3 countries: United States, Italy, Taiwan
Source: ClinicalTrials.gov record NCT04327973. Inclusion in this directory is not an endorsement.