Observational study of outcomes in cystic fibrosis patients with selected gating mutations


Therapeutic category
Trial status
closed Participating Centres
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 Reference Number
Trial type
Length of participation
48 months
CFTR Modulator (Ivacaftor)
Last edited date
CF sponsor
Vertex Pharmaceuticals Incorporated
CF sponsor type

Who can take part?

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

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