IMPORTANT INFORMATION

  • CFTR.info is an online educational resource on the genetics of cystic fibrosis (CF) and the role of CF transmembrane conductance regulator (CFTR) in normal human physiology as well as in CF.
  • The information contained herein is intended for healthcare professionals working within the European Union (EU).
  • The content of this website has been developed independently by a group of CF healthcare professionals and is for general information purposes only.
  • All of the information about CFTR and CFTR modulators has been obtained from referenced sources and is in the public domain.
  • This website is not intended to promote or advocate a particular course of treatment or any specific drug, whether licensed or unlicensed, nor is it intended to imply or suggest that any drug be used for any purpose other than that which for which it is licensed in the EU.

ABBREVIATED TERMS & CONDITIONS

  • While we endeavour to keep the information up to date and correct, we make no representations or warranties of any kind, express or implied, about the completeness, accuracy, reliability, suitability or availability with respect to the website or the information and related graphics, or products and treatments, contained on the website for any purpose. Any reliance you place on such information is therefore strictly at your own risk.
  • In no event will we be liable for any loss or damage including without limitation, indirect or consequential loss or damage, or any loss or damage whatsoever arising from, or in connection with, the use of this website.
  • Through this website you are able to link to other websites which are not under our control. We have no control over the nature, content and availability of those sites. The inclusion of any links does not necessarily imply a recommendation or endorse the views expressed within them.
  • Every effort is made to keep the website up and running smoothly. However, we take no responsibility for, and will not be liable for, the website being temporarily unavailable due to technical issues beyond our control.

Please see the full Terms & Conditions before using this website.

 

I have read and understood the above

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

Click on the tabs to move through the sequence.

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

Intervention
Ivacaftor

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

Click on the tabs to move through the sequence.

  • 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