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
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
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
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Length of participation
the length of time a participant will take part in a trial, from the first to the last appointment
Last edited date
05 September 2017
Vertex Pharmaceuticals Incorporated
CF sponsor type