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VX14770116: Ph IV Observational Study for CF Patients


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


Trial status


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 IV

Full title

Observational Study of Outcomes in Cystic Fibrosis Patients with Selected Gating Mutations on a CFTR Allele (The VOCAL Study)

The study is being done to learn more about the effectiveness of Kalydeco in CF patients with the following specific gene mutaions: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. The study also looks to explore the effect of Kalydeco on your quality of life. This is an observational study. This means that your cystic fibrosis treatment and medical care will not change because you are participating in this study and the sponsor will not pay for Kalydeco. Your doctor will continue to make all decisions regarding your proper treatment and care. No part of this study is experimental.

Trial type


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

48 months

Last edited date

05 September 2017


Vertex Pharmaceuticals Incorporated

Sponsor type


Who can take part?


No copies of F508del


0 - 12

Top inclusion criteria
  • 6 years of age +
  • Male or female with confirmed diagnosis of cystic fibrosis
  • At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D
Top exclusion criteria
  • Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment
  • Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation
  • History of organ transplantation

CF centres running this trial


Heart of England NHS Foundation Trust


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

Recruitment ends

July 2020


Nash, Edward

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Papworth Hospital NHS Foundation Trust


Papworth Hall Papworth Everard Cambridge Cambridgeshire CB23 3RE

Recruitment starts

July 2020



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Royal Brompton and Harefield NHS Foundation Trust


Royal Brompton Hospital Sydney Street London Greater London SW3 6NP

Recruitment starts

February 2016

Recruitment ends

August 2016


Simmonds, Nicholas

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