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VX15-770-124 Cystic Fibrosis


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 at Treatment Initiation and Have a CFTR Gating Mutation

This is a Phase 3, 2-part, open-label study designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in subjects with CF who are <24 months of age at treatment initiation and have a CFTR gating mutation on at least 1 allele. Part A is designed to evaluate the safety and PK of multiple-dose administration of ivacaftor in subjects <24 months of age and to confirm (or adjust if necessary) the doses for Part B. Part B is designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in subjects <24 months of age over 24 weeks.

Trial Reference Number


Trial type


the name of the treatment or therapy being researched


Recruitment target
the number of participants who need to be recruited for the trial in the UK


Last edited date

09 October 2017

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type


Who can take part?


0 - 12

Top inclusion criteria
  • Aged 0 - 24 months
  • Male or female with confirmed diagnosis of CF
  • Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
Top exclusion criteria
  • History of solid organ or hematological transplantation
  • Colonization with organisms associated with a more rapid decline in pulmonary status (e.g., Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus) at screening
  • History of abnormal liver function or abnormal liver function at screening

CF centres running this trial


Great Ormond Street Hospital


Great Ormond Street London WC1N 3JH

Recruitment starts

February 2017

Recruitment ends

February 2020


Aurora, Paul

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University Hospitals of Leicester NHS Trust


University Hospitals of Leicester Headquarters Level 3 Balmoral Building Leicester Royal Infirnary Infirmary Square Leicester Leicestershire LE1 5WW

Recruitment starts

October 2016

Recruitment ends

June 2019


Gaillard, Erol

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Central Manchester University Hospitals NHS Foundation Trust


Trust Headquarters Cobbett House Manchester Royal Infirmary Oxford Road Manchester Greater Manchester M13 9WL

Recruitment ends

June 2018


Maitra, Anirban

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Royal Brompton and Harefield NHS Foundation Trust


Royal Brompton Hospital Sydney Street London Greater London SW3 6NP

Recruitment starts

September 2016

Recruitment ends

May 2018


Davies, Jane C

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Show all participating centres