drugset / Trial / NCT04094948
Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The goals of this study are to determine safety and efficacy with regard to motor function of oral clenbuterol in combination with ERT in subjects with LOPD
Timeline
- Start
- 2023-01-01
- Primary completion
- 2025-06-30
- Completion
- 2025-12-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Clenbuterol | Small molecule | 80 ug | Oral |