drugset / Trial / NCT01580501
PDE Inhibitors in DMD Study (Acute Dosing Study)
NaSingle-groupOpen-labelTreatment
Summary
PDE5A inhibition, which boosts NO-cGMP signaling, will relieve functional muscle ischemia and restore normal blood flow regulation (i.e., functional sympatholysis) during exercise in boys with DMD. The investigators specific aim is to perform an efficient dose-titration study to inform the design of a randomized multicenter trial of PDE5A inhibition for clinical skeletal muscle and cardiac endpoints.
Timeline
- Start
- 2012-03
- Primary completion
- 2013-03
- Completion
- —
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Sildenafil | Unknown | 0.5 mg/kg | — |
| Subject | Sildenafil | Unknown | 1 mg/kg | — |
| Subject | Tadalafil | Small molecule | 0.5 mg/kg | — |
| Subject | Tadalafil | Small molecule | 1 mg/kg | — |