drugset / Trial / NCT01556568
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of MEK162 in Noonan Syndrome Hypertrophic Cardiomyopathy
NaSingle-groupOpen-labelTreatment
Summary
The purpose of the study is to determine whether the ability of MEK162 to antagonize MEK activation in NS HCM patients, who usually have upstream mutations in the Ras-Raf-Mek-Erk pathway that lead to MEK activation, would be beneficial over a 6 month treatment period in hypertrophy regression.
Timeline
- Start
- 2012-02
- Primary completion
- 2017-05
- Completion
- —
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Binimetinib | Small molecule | — | — |
Indications
No indication recorded.