Effect of inhaled Nacystelyn® on the incidence of recurring PEs in CF

• Research type

  Research Study

• Full title

  A Phase III, Multicentre, Randomised, Placebo-Controlled, Double Blind Study of the Incidence of Recurring Pulmonary Exacerbations in Cystic Fibrosis Patients using Two Different Doses of Inhaled Nacystelyn®

• IRAS ID

  53888

• Contact name

  Martin Ledson

• Sponsor organisation

  Laboratoires SMB S.A.

• Eudract number

  2010-018454-13

• Research summary

  Cystic Fibrosis (CF) is the most common life-shortening genetic disorder in Caucasians and affects approximately 30,000 people in Europe. Current management of the condition involves easing the symptoms and slowing the progress of the disease. CF patients are very susceptible to frequent lung infections which cause breathing difficulties and over time, a decline in lung function. This is the primary cause of hospitalizations in CF patients.The drug under study in this trial (NAL) is a combination of two drugs which are well established and widely available. NAL has been used orally for many years to help clear the lungs. NAL has now been developed by the Pharmaceutical company funding this study to be taken as a dry powder inhaler (DPI) and patients will be dosed twice daily for a total of 52 weeks. The study will be run in 13 countries and over 530 patients will take part. There will be a 2 week run-in period before the treatment starts for the patients to become used to the Inhaler device. Patients will then be randomly allocated to one of three treatment groups; high dose NAL, low dose NAL or placebo. The placebo group will allow for detection of false treatment effects. Approximately half the patients recruited onto the study will also be receiving another drug called rhDNase to allow comparison of NAL in the presence and absence of this drug. Males and females over 12 years old with a confirmed diagnosis of CF will be allowed to take part in the study. There will be 8 visits to the hospital clinics during the 52 week treatment period at which lung function and safety measurements will be taken to assess the long term safety and efficacy of NAL in the presence and absence of a mucolytic.

• REC name

  HSC REC B

• REC reference

  10/NIR03/34

• Date of REC Opinion

  1 Sep 2010

• REC opinion

  Further Information Favourable Opinion