Safety & Efficacy of VX-440 Combination Therapy for Cystic Fibrosis
Research type
Research Study
Full title
A Phase 2, Randomized, Double-blind, Controlled Study to Evaluate the Safety and Efficacy of VX-440 Combination Therapy in Subjects Aged 12 Years and Older With Cystic Fibrosis
IRAS ID
207883
Contact name
Jane Davies
Contact email
Sponsor organisation
Vertex Pharmaceuticals Incorporated
Eudract number
2016-000454-36
Clinicaltrials.gov Identifier
129,421, IND Number
Duration of Study in the UK
1 years, 2 months, 27 days
Research summary
The purpose of this study is to find out about the safety and efficacy of VX-440 when administered in combination with two other medications (tezacaftor (TEZ) and ivacaftor (IVA)) for the treatment of Cystic Fibrosis.
VX-440 and TEZ are CFTR correctors; they increase chloride ion [CL-] secretion by improving processing and trafficking. They are not approved by the US, EU or UK Health Authorities (FDA, EMA, MHRA), so are considered investigational.
IVA is a CFTR potentiator, which increases chloride ion [CL-] secretion by making it more likely for the ion channel to be open. IVA is approved for the treatment, for certain CF mutations, in a number of countries globally for use in patients with CF as young as 2 years.
There are three parts in this study. Around 198 people worldwide, with two types of Cystic Fibrosis-causing mutations, will take part. Around 32 in Part 1, 24 in Part 2 and 132 in Part 4 (of which at least 30 will be children between 12-17 years). People in Part 1 will be in the study for around 12 weeks, in Parts 2 and 4 participation will last around 20 weeks. Participants might be in the study longer if they roll over from another Vertex study.
Procedures will include medical and demographic questionnaires, body exam, vital sign measurements, pulse oximetry, eye exam, ECG (electrocardiogram), sweat chloride test, spirometry, urine test, pregnancy test, DNA test (Cystic Fibrosis gene analysis), and blood tests.
This is a global study that will run is about 50 medical canters in 12 countries. Vertex Pharmaceuticals is funding the research.
REC name
London - Hampstead Research Ethics Committee
REC reference
16/LO/1875
Date of REC Opinion
10 Jan 2017
REC opinion
Further Information Favourable Opinion