drugset / Trial / NCT00688597
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
Non-randomizedParallel-groupOpen-labelTreatment
Summary
The main purpose of this study was to determine the safety and tolerability of 3 different doses of duvoglustat (AT2220) in participants affected by Pompe disease. The study also evaluated the effects of duvoglustat on functional parameters in Pompe disease.
Timeline
- Start
- 2008-12-08
- Primary completion
- 2009-12-14
- Completion
- 2009-12-14
Outcome
Outcome not reported
Stopped: “Per protocol, 3 sequential dose cohorts were planned. Study discontinued by Sponsor based upon serious adverse events in first 2 of 3 participants in Cohort 1.”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Duvoglustat | Small molecule | 2.5 g | Oral |
| Subject | Duvoglustat | Small molecule | 5 g | Oral |