A study of VX-770 in Subjects aged 6 to 11 with Cystic Fibrosis

• Research type

  Research Study

• Full title

  A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Pharmacokinetics, Efficacy and Safety of VX-770 in Subjects Aged 6 to 11 Years with Cystic Fibrosis and the G551D Mutation

• IRAS ID

  24901

• Eudract number

  2008-007479-26

• ISRCTN Number

  n/a

• Research summary

  Vertex Pharmaceuticals Incorporated is developing a drug called VX-770 as a possible treatment for Cystic Fibrosis (CF). VX-770 is an experimental drug, which means that it has not been approved for prescribing to people. VX-770 is being studied to see if it can help decrease some of the effects caused by CF. This study is also being done to learn more about the safety and effects of VX-770 on CF patients. This is a randomized, double-blind and placebo-controlled study. Randomized means that the choice of whether the patient will be given VX-770 or a pill that looks just like VX-770, but does not contain any medicine ( a placebo), will be assigned by a computer, by chance, like the flip of a coin. They will have a 50:50 chance of receiving VX-770. No one can choose what anyone receives. Double-Blind means that no one at the study site will know if the patient is taking VX-770 or the pill that does not contain any medication. The doctor can find out what medication the patient is on if it is necessary. Placebo controlled means that a pill that looks just like the VX-770 pill but does not contain VX-770 or any other medicine (a placebo) will be taken by some people in this study. This is done so that the study findings can be looked at fairly.This study will check the safety and effect of taking 100 mg of VX-770 by mouth twice a day for 48 weeks. Study drug levels in blood will also be checked. Taking part in this part of the study is expected to last up to 3 years.

• REC name

  South Central - Hampshire B Research Ethics Committee

• REC reference

  09/H0504/82

• Date of REC Opinion

  27 Jul 2009

• REC opinion

  Further Information Favourable Opinion