First patient in the world recruited to new Cystic Fibrosis CFTR modulator therapy trial

A clinical trial conducted by the Cystic Fibrosis team at Manchester Adult Cystic Fibrosis Centre (MACFC) is underway. The first participant in the world has been recruited to the VX18-121-101 research study at the NIHR Manchester Clinical Research Facility (CRF) at Wythenshawe Hospital.

Cystic fibrosis (CF) is the commonest rare disease, with more than 10,400 people affected in the UK. CF is a disease that affects many different organs, and causes thick and sticky mucus to build up in the lungs and digestive system. This increased mucus can cause blockages in the lung, which make it difficult to breathe and allows the lungs to become infected. Current treatment includes a range of medications to help people breathe better, prevent infections, and improve nutrition.

A person will be born with CF when the faulty cystic fibrosis transmembrane conductance regulator (CFTR) gene from both parents is genetically passed on. CFTR modulator therapies target the faulty protein made by the CFTR gene. There are more than 1900 different variants of the CFTR gene, but most people with CF (over 90% in the UK) have at least one copy of the faulty F508del gene.

The VX18-121-101 study aims to test if a new oral medication, the CFTR corrector “VX-121”, is safe and works as expected when taken in with two other medications: tezacaftor (TEZ) and ivacaftor (IVA), in people with cystic fibrosis aged 18 years and over.


vx121-a

L-R: CTAP Clinical Trial Coordinator Natalie Hill; CF Specialist Clinical and Research Nurse Cassie MacNaughton; 1st global patient; CF Consultant and European Chief Investigator Dr Alex Horsley and Manchester Clinical Research Facility Research Nurse Sian Leech

The aim of the trial is to help researchers understand if this triple drug treatment improves salt transport (to make mucus less thick) in two groups of people: those with one copy of the F508del gene, and those with two copies. This trial is being led by Dr Alex Horsley, CF Consultant at MACFC, Wythenshawe Hospital; Reader in respiratory medicine at the University of Manchester and Medical Director, Manchester CRF at Wythenshawe Hospital.

This is a very exciting time for CF research. If the trials are successful these drugs could provide a really important treatment for most of our CF patients. We are proud and privileged as a team to have been involved with this cutting edge research in cystic fibrosis.

Dr Alex Horsley

Dr Horsley is European lead for this programme, and this project forms part of a wider portfolio of Cystic Fibrosis research being conducted at MACFC. This research has been supported by the National Institute for Health Research Clinical Research Network: Greater Manchester, and is sponsored and funded by Vertex Pharmaceuticals Inc.