Investigation into the efficacy of MEK162 in Noonan syndrome V1

• Research type

  Research Study

• Full title

  An open label study to assess safety, tolerability, pharmacokinetics and pharmacodynamics of MEK162 in Noonan syndrome hypertrophic cardiomyopathy

• IRAS ID

  87471

• Contact name

  William McKenna

• Sponsor organisation

  Novartis

• Eudract number

  2011-003392-10

• ISRCTN Number

  isrctn

• Clinicaltrials.gov Identifier

  nct

• Research summary

  Hypertrophic cardiomyopathy (HCM) affects about 1 in 500 individuals. Individuals affected with HCM may not have any symptoms or may experience shortness of breath and angina, and these patients are at risk for heart failure, arrhythmias of the heart and sudden death. Possible treatment options for HCM patients are either for symptom relief or towards prevention of sudden death. However, none of these options are completely effective, and disease and death rates remain high. There remains a major unmet medical need in these patients for both symptom relief and patient survival. Noonan syndrome (NS) is a common cause of HCM. Patients with NS have increased cardiomyopathy (increase in thickness of heart muscle). If the thickness of the heart muscle can be reduced it may decrease the disease and death rates. NS is caused by genetic defects which cause a protein called MEK to be activated. The purpose of this study is to determine whether the ability of the drug under investigation to antagonize the activation of MEK in these patients would be beneficial over a 6 month treatment period by causing reduced enlargement of the heart muscle. This study will be carried out in two centres, involving 22 patients with NS who will take the drug for 6 months and undergo assessment of heart muscle thickness, safety of the drug and blood levels of the drug.

• REC name

  London - Harrow Research Ethics Committee

• REC reference

  11/LO/1773

• Date of REC Opinion

  23 Jan 2012

• REC opinion

  Further Information Favourable Opinion