VX11-661-101 A Study Evaluating VX-661 & VX-661/770 in CF Patients

• Research type

  Research Study

• Full title

  A Phase 2, multicenter, double-blinded, placebo-controlled, 3-part study to evaluate safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of VX-661 monotherapy and VX-661/VX-770 cotherapy in subjects with cystic fibrosis, homozygous for the F508del-cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation

• IRAS ID

  96959

• Contact name

  Jane Davies

• Sponsor organisation

  Vertex Pharmaceuticals Incorporated

• Eudract number

  2011-003821-93

• Clinicaltrials.gov Identifier

  n/A

• Research summary

  This is a 3-part study looking at the safety, tolerability and effectiveness of the study drugs VX-661 and VX-770 in adults with cystic fibrosis (CF) who have the F508del-CFTR gene mutation. Patients will be enrolled into a treatment group in one of the 3 parts; A, B or C. In each part, patients will be placed at random into different treatment groups to receive, either, VX-661, VX-661 plus VX-770 or placebo.There will be around 120 patients in Part A and 80 in Part B. Enrolment in Part B may be initiated after enrollment in Part A has been completed. The doses used Part B will depend on results from Part A. Both parts of the study will include a Screening Period, a 28-day Treatment Period, outpatient visits on Study Days 35 and 42, and a Follow-up Visit. The study drug(s) will be taken inpatient (at the clinical site) or outpatient (outside the clinical site), as applicable, during the Treatment Period. The daily dose of VX-661 will be administered with the morning dose of VX-770. The last dose of study drug(s) in each part of the study will be the morning dose on Study Day 28. Initiation of Part C may occur at any time after the first interim analysis in Part A is completed. In Part C, 4 to 8 subjects will receive 1 dose level of VX-661. The dose level in Part C will be based on results from Part A. Similar to the design of Parts A and B, there will be 28 days of treatment and a 28-day washout.

• REC name

  London - Riverside Research Ethics Committee

• REC reference

  12/LO/0321

• Date of REC Opinion

  1 May 2012

• REC opinion

  Further Information Favourable Opinion