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A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation


Therapeutic category
the type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise

Restore CFTR Function

Trial status

Open to recruitment

Participating Centres
the different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use

Phase III

Full 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 Less Than 24 Months of Age and Have a CFTR Gating Mutation

The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have a CF transmembrane conductance regulator (CFTR) gene gating mutation

Trial Reference Number


Trial type


Length of participation
the length of time a participant will take part in a trial, from the first to the last appointment

104 weeks

the name of the treatment or therapy being researched


Last edited date

14 December 2017

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type


Who can take part?

Top inclusion criteria
  • Subjects transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
  • One of the following CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. Subjects who have an R117H-CFTR mutation will be eligible in regions where ivacaftor is approved for use in subjects 2 through 5 years of age with an R117H-CFTR mutation.
Top exclusion criteria
  • Subjects receiving commercially available ivacaftor treatment

CF centres running this trial


Great Ormond Street Hospital


Great Ormond Street London WC1N 3JH

Recruitment ends

February 2019



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Royal Hospital for Sick Children Edinburgh


Waverley Gate 2-4 Waterloo Place Edinburgh EH1 3EG

Recruitment ends

November 2018



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Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust


Oxford Road,Manchester, M13 9WL

Recruitment ends

May 2019


Anirban Maitra

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