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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 an Approved Ivacaftor-Responsive Mutation

Details

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
Phase
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-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation

This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation

Trial Reference Number

105223

Trial type

Medication

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

104 weeks

Intervention
the name of the treatment or therapy being researched

Ivacaftor

Last edited date

05 June 2019

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type

Commercial

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.
  • An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible in countries/regions where ivacaftor is approved for use in subjects 2 years of age and older
Top exclusion criteria
  • Subjects receiving commercially available ivacaftor treatment

CF centres running this trial

Closed

Alder Hey Children's Hospital

Address

Alder Hey Hospital Eaton Road West Derby Liverpool Merseyside L12 2AP

Recruitment starts

July 2019

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Closed

Great Ormond Street Hospital

Address

Great Ormond Street London WC1N 3JH

Recruitment ends

February 2019

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Closed

John Radcliffe Hospital

Address

HeadingtonHeadley WayOxfordHeadley WayOxfordshire OX3 9DU

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Closed

Royal Hospital for Sick Children Edinburgh

Address

Waverley Gate 2-4 Waterloo Place Edinburgh EH1 3EG

Recruitment ends

November 2018

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