drugset / Trial / NCT01099761
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The purpose of this study is to determine if ACE-031 is safe and well-tolerated in boys with Duchenne Muscular Dystrophy (DMD) and to select the optimal doses of ACE-031 in terms of safety and pharmacodynamic (PD) activity for designing future studies. \[Note: This study was terminated based on safety data\]
Timeline
- Start
- 2010-04
- Primary completion
- 2011-06
- Completion
- 2011-06
Outcome
Outcome not reported
Stopped: “Based on preliminary safety data.”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | RAMATERCEPT | Protein / enzyme biologic | 0.5 mg/kg | Subcutaneous |
| Subject | RAMATERCEPT | Protein / enzyme biologic | 1 mg/kg | Subcutaneous |