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Drug & research development » Clinical trials » NCT01931839

A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis

Official title: A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation

Clinical Trials gov number: NCT01931839

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Purpose: To evaluate the efficacy and safety of long-term treatment with VX-809 in combination with ivacaftor in CF patients aged ≥12 years.

Phase: 3

Type: Interventional, randomized, double-blind, parallel assignment

Study sponsor: Vertex Pharmaceuticals Incorporated

  • Signed informed consent form (ICF), and where appropriate, signed assent form
  • Subjects entering the Part A Treatment Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104 and elect to enroll in Part A treatment cohort
  • Subjects entering the Part B Treatment Cohort: Completed 56 days of study drug treatment in Cohort 4 of Study 102 and elect to enroll in Part B treatment cohort
  • Subjects entering the Part A Observational Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104, but do not elect to enroll in the Part A Treatment Cohort or do not qualify to enroll in Part A treatment cohort
  • Willing to remain on a stable CF medication regimen through the end of study (Part A and Part B Treatment Cohorts only)

Intervention
Ivacaftor, Lumacaftor

Geographical Location
Australia, Austria, Belgium, Canada, Czech Republic, Denmark, France, Germany, Italy, Ireland, Netherlands, Spain, Sweden, United Kingdom, United States

Number of Participants
≥1001 (≥12 years)

Primary Endpoint

Safety based on adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms, vital signs, and pulse oximetry to Week 100

Secondary Endpoint

Baseline to 100 weeks
· Absolute change from baseline in percent predicted FEV1
· Relative change from baseline in percent predicted FEV1
· Rate of change in percent predicted FEV1
· Absolute change from baseline in Cystic Fibrosis Questionnaire – Revised (CFQ-R) respiratory domain score
· Absolute change from baseline in body mass index (BMI)
· Absolute change from baseline in body weight

Baseline to 100 weeks (Part A only)
· Absolute change in BMI z score
· Number of pulmonary exacerbations starting from the previous study, and in the current study
Time to first pulmonary exacerbation, including pulmonary exacerbations in the previous study, and in the current study
· Event of having at least 1 pulmonary exacerbation, including pulmonary exacerbations in the previous study, and in the current study

Up to 2 years
· Part A Observational Cohort: Safety, as determined by Serious Adverse Events

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  • Experimental: Part A Treatment Cohort

Arm 1: 600 mg lumacaftor once daily (qd) + 250 mg ivacaftor every 12 hours (q12h)

Arm 2: 400 mg lumacaftor q12h + 250 mg ivacaftor q12h

  • Experimental: Part B Treatment Cohort

Arm 3  400 mg lumacaftor q12h + 250 mg ivacaftor q12h

  • No Intervention: Part A Observational Cohort

Long-term Follow-up

View Trial Results