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Drug & research development » Clinical trials » NCT01705145

Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CTFR Gating Mutation

Official title: A Phase 3, 2-Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are 2 Through 5 Years of Age and Have a CFTR Gating Mutation

Clinical Trials gov number: NCT01705145

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Purpose: To evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD), of ivacaftor in children with CF who are aged 2–5 years and have a CFTR gating mutation in ≥1 allele.

Phase: 3

Type: Interventional, open-label, single group assignment

Study sponsor: Vertex Pharmaceuticals Incorporated; Cystic Fibrosis Foundation Therapeutics

  • Male or female with confirmed diagnosis of CF
  • CFTR gating mutation in ≥1 allele
  • Aged 2–5 years at screening and Day 1
  • Weight ≥8kg at screening and Day 1
  • Haematology, serum chemistry, coagulation, and vital signs results at screening with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator


Geographical Location
Canada, United Kingdom, United States

Number of Participants
≤50 (2-5 years)

Primary Endpoint

Baseline to Week 24

  • Safety, as determined by adverse events, clinical laboratory values (serum chemistry, hematology, and coagulation studies), electrocardiograms, vital signs and ophthalmologic examinations

Secondary Endpoint

Baseline (BL) to Week 24:

  • PK parameter (Cmin and AUC) estimation of ivacaftor and metabolite
  • Absolute change from BL in: sweat chloride; weight; stature and body mass index

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  • Single Arm

Part A: 50 mg (for subjects <14 kg) or 75 mg (for subjects ≥14 kg) ivacaftor will be administered every 12 hours (q12h) on Days 1 through 3, and 1 morning dose on Day 4

Part B: 50 mg (for subjects <14 kg) and 75 mg (for subjects ≥14 kg) ivacaftor will be administered q12h for 24 weeks

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