Observational study of outcomes in cystic fibrosis patients with selected gating mutations
Details
- Therapeutic approach
- Other
- Trial status
- Closed with results Participating Centres
- Trials Tracker ID
- TT001606
- Last updated
- 16/10/2019
Observational Study of Outcomes in Cystic Fibrosis Patients with Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
Study detailsThe 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.
- Phase
- Phase 4
- Length of participation
- 48 months
- CF sponsor
- Vertex Pharmaceuticals Incorporated
- CF sponsor type
- Commercial
Who can take part?
- Age range
- 6 years and older
- Including people
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- Excluding people
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
Get in touch