New CF Studies at Edinburgh Children's CRF

Vertex VX11-770-110 (and 112)

Vertex 110 study is a study of Ivacaftor in cystic fibrosis patients with a R117H gene mutation. In the UK this is approximately 4-5% of all CF patients. R117H is a class four CF mutation which in the laboratory demonstrates benefit from Ivacaftor. This double blind randomised controlled trial in adults and children over 6 years of age is recruiting to the 110, which once completed will roll over into the open label (not blinded) 112 study. Participant from across Scotland are contributing to this study attending the Edinburgh Children’s CRF.

Vertex VX11-770-108 (and 109)

Vertex 108 study is a study of Ivacaftor in cystic fibrosis patients with a G551D gene mutation. In the UK this is approximately 4-5% of all CF patients. G551D is a class three CF mutation which in trial in adults and children over 6 years of age demonstrates very good effect from Ivacaftor (Kalydeco – licensed in Europe and USA). This open label trial in children between the ages of 2-5 years of age is recruiting to the 108 study, which once completed will roll over into the 109 continuation study. Participant from across Scotland are contributing to this study attending the Edinburgh Children’s CRF.

TORPEDO

TORPEDO is an HTA funded UK multicentre study wishing to assess the optimal treatment for growth of Pseudomonas aeruginosa in respiratory secretions in patients with Cystic Fibrosis. Patients are randomly allocated to either two weeks of intravenous antibiotics (Ceftazidime and Tobramycin) or three months of oral ciproxin (both treatment arms have 3 months of nebulised Colistin). Patients isolating pseudomonas aeruginosa in Edinburgh are being invited to participate in the trial which is being co-ordinated by the Medicines for Children Research Network in Liverpool and Dr Simon Langton-Hewer in Bristol in Chief Investigatory. The study is approximately half way to its recruitment target in Summer 2013.

Contact

If you are interested in learning more about these studies please contact Steve.Cunningham@nhslothian.scot.nhs.uk or kayriding@nhslothian.scot.nhs.uk