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A Phase 1/2 Study of VX-121 in Subjects With Cystic Fibrosis and in Subjects without 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

Closed to recruitment - in follow up

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 I/II

Full title

A Phase 1/2 Study of VX-121 in Subjects With Cystic Fibrosis and in Subjects without Cystic Fibrosis

The purpose of this study is to evaluate safety and tolerability of VX-121 in healthy subjects and in subjects with cystic fibrosis.

Trial Reference Number


Trial type


the name of the treatment or therapy being researched

CFTR Modulators (Ivacaftor / Tezacaftor)

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


Last edited date

26 June 2019

CF sponsor

Vertex Pharmaceuticals Incorporated

CF sponsor type


Who can take part?


One copy of F508del



Top inclusion criteria
  • One copy of F508del mutation
  • Body weight ≥35 kg
  • Must be able to produce a sweat sample at screening
Top exclusion criteria
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for sinopulmonary disease
  • History of cirrhosis (liver injury)
  • Risk factors for Torsade de Pointes (abnormal heart rhythm) and other ventricular arrhythmias (abnormal heartbeats)

CF centres running this trial


Heart of England NHS Foundation Trust


Birmingham Heartlands Hospital Bordesley Green East Birmingham West Midlands B9 5SS

Recruitment starts

December 2018

Recruitment ends

February 2019


Nash, Edward

Get in touch


Medicines Evaluation Unit


The Langley Building Southmoor Road Wythenshawe Manchester M23 9QZ


Horsley, Alexander

Get in touch