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Drug & research development » Clinical trials » NCT01946412

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

Official title: A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric Subjects With Cystic Fibrosis and a CFTR Gating Mutation

Clinical Trials gov number: NCT01946412

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Purpose: To provide information on the long-term safety and pharmacodynamics of ivacaftor treatment in CF patients aged <6 years who have a CFTR gating mutation in ≥1 allele. To further explore the efficacy of long-term ivacaftor treatment in this population of CF patients.

Phase: 3

Type: Interventional, non-randomized, open-label, parallel assignment

Study sponsor: Vertex Pharmaceuticals Incorporated; Cystic Fibrosis Foundation Therapeutics

Ivacaftor arm:

– Completed the last study visit of the treatment period of the previous study (NCT01705145)

– Haematology, serum chemistry, and vital signs results on Day 1 with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator

– As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions and the parent or legal guardian should be able to ensure that the subject assents to participation in the study to the degree the subject can assent, and that the subject will comply with and is likely to complete the study as planned

Observational arm:

– Subjects who completed their assigned study drug treatment in the previous study (NCT01705145) and elected not to enrol in the ivacaftor arm

– Subjects who prematurely discontinued treatment in the previous study and received ≥1 dose of study drug treatment in the previous study


Geographical Location
Canada, United Kingdom, United States

Number of Participants
≤50 (≥2 years)

Primary Endpoint

Baseline to Week 88

  • Safety, as determined by adverse events, clinical laboratory values (serum chemistry and hematology), standard 12-lead electrocardiograms, vital signs and ophthalmologic examinations

Secondary Endpoint

Absolute change to Week 88 from previous study baseline (BL) and from BL in:
· Weight
· Stature
· Body mass index (BMI)
· Sweat chloride

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  • Observational arm: no intervention
  • Experimental arm: Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the Week 84 Visit.

The ivacaftor dose will be: 50 mg q12h for subjects 2 to <6 years of age and <14 kg, 75 mg q12h for subjects 2 to <6 years of age and ≥14 kg, or 150 mg q12h for subjects ≥6 years of age

View Trial Results