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Drug & research development » Clinical trials » NCT01807949

A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation (TRANSPORT)

Official title: A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Clinical Trials gov number: NCT01807949

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Purpose: To evaluate the efficacy and safety of Lumacaftor in combination with ivacaftor in CF patients aged ≥12 years who are homozygous for the F508del mutation.

Phase: 3

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

Study sponsor: Vertex Pharmaceuticals Incorporated

  • Males and females, aged 12 years or older on the date of informed consent or, where appropriate, date of assent
  • Confirmed diagnosis of CF
  • Homozygous for the F508del CFTR mutation
  • FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
  • Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the safety follow-up visit

Intervention
Ivacaftor, Lumacaftor

Geographical Location
Australia, Austria, Belgium, Canada, Denmark, France, Germany, Spain, United Kingdom, United States

Number of Participants
101-1000 (12-65 years)

Primary Endpoint

Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) from baseline at Week 24

Secondary Endpoint

From baseline at Week 24

  • Relative change in percent predicted FEV1
  • Absolute change in body mass index
  • Absolute change in Cystic Fibrosis Questionnaire – Revised (CFQ R) respiratory domain score
  • Absolute change in BMI z-score
  • Absolute change in body weight
  • Absolute change in EuroQol 3 Level (EQ 5D 3L)
  • Absolute change in Treatment Satisfaction Questionnaire for Medication (TSQM) domains

To Week 24

  • Number of pulmonary exacerbations
  • Event of having at least 1 pulmonary exacerbation
  • Time-to-first pulmonary exacerbation

Week 28

  • Safety and tolerability assessments based on adverse events, clinical laboratory values (hematology, serum chemistry, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), ambulatory ECGs, vital signs, and pulse oximetry

Week 16

  • PK parameters of lumacaftor, M28 lumacaftor, ivacaftor, M1 ivacaftor, and M6 ivacaftor

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  • Experimental treatment arm 1

Subjects randomized to study drug will take 600 mg of lumacaftor once daily (qd) and 250 mg of ivacaftor every 12 hours (q12h)

  • Experimental treatment arm 2

Subjects randomized to study drug will take 400 mg of lumacaftor every 12 hours (q12h) and 250 mg of ivacaftor every 12 hours (q12h)

  • Experimental treatment arm 3

Subjects randomized to placebo will remain on placebo through 24 weeks. Subjects will be given tablets that match both lumacaftor and ivacaftor and will follow the same dosing regimen as subjects receiving the study drug

View Trial Results