Trials Tracker

Due to COVID-19 some cystic fibrosis (CF) clinical trials may be still be temporarily paused, however some trials will begin to re-open soon. If you have any questions about taking part in clinical trials, please speak to our CF team or contact the Trust’s clinical trial team. Please also see the relevant advice on the COVID-19 Q&A page. The Trials Tracker brings together all CF trials currently recruiting in the UK, so you can find clinical trials you can take part in both in your region and further afield

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A study of the combined effects of three CFTR modulator medicines in children with cystic fibrosis aged 6 to 11 (VX18-445-106)

VX-445 is an investigational medicine being studied to see how well it works and how safe it is in children with cystic fibrosis aged 6 to 11 years, when taken in combination with tezacaftor and ivacaftor. All three medicines are CFTR modulators which means that they help the faulty CFTR protein found in cystic fibrosis to work properly. Tezacaftor is already approved for use and is what is known as a ‘corrector’. Ivacaftor is also already approved for use and is what is known as a ‘potentiator’. VX-445 is a corrector. Trial participants will be asked to take VX-445, tezacaftor and ivacaftor in the morning (2 tablets), and ivacaftor in the evening (1 tablet). Everyone in the study will receive all three of the study medications, no placebo is involved. This study is divided into two parts. Part A will recruit 12 people who will be asked to come into clinic around 6 times over 10 weeks. Part B will recruit 56 people who will be asked to come into clinic around 11 times over 32 weeks. During visits, the effect of the treatment on your CF will be measured with sample collection, lung function, questionnaires, and other medical checks.

CF START

CF START is a national UK trial that will determine the safest and most effective antibiotic strategy for infants diagnosed with cystic fibrosis (CF). 480 CF infants will be randomly allocated either flucloxacillin prophylaxis (the current UK standard of care) or antibiotics given in a more targeted manner. The primary outcome will be the age at first growth of Pseudomonas aeruginosa from a respiratory culture (an important safety measure for families). All outcomes will be recorded on a national CF Registry, including a number of secondary outcomes assessing effectiveness and safety. Finally at 40-48 months, a measure of respiratory function will be undertaken in a central laboratory, which will provide a clearer indication of the effectiveness of these two strategies.

A Study to Assess various doses of an ENaC inhibitor medication (mucociliary clearance) in Healthy Volunteers and People with Cystic Fibrosis

A study medication called ION-827359 is being developed to treat lung disease symptoms in people with cystic fibrosis. The study medication is intended to help treat the thick mucus found in the airways of patients with cystic fibrosis. This new inhaled (nebulized) medication will be investigated in both people with, and without cystic fibrosis. The study will compare the study medication with a placebo (a solution that looks identical to the study medication but contains no active ingredients), which means some participants will receive the study medication, whilst others will receive the placebo. Participants won’t know whether they are receiving the study medication or the placebo. Participation in the study lasts for approximately 21 weeks (just over 5 months), and will require numerous visits to complete study assessments (blood tests, questionnaires, lung function etc.) to the recruitment centre.

High Flow Nasal Therapy during exercise in CF

High flow nasal therapy (HFNT) is a way to deliver a mixture of air and oxygen at higher flows compared to standard oxygen therapy. It has been shown to improve oxygenation, reduce breathlessness and breathing rate, and is quite comfortable. Recently HFNT has been shown to improve exercise capacity and tolerance in patients with other lung diseases. HFNT is routinely used in clinical practice in patients with CF who present acutely unwell during hospital admissions. We believe that HFNT could be used by patients with CF during exercise in order to improve their tolerance, lengthen their exercise sessions, and allow them to be less symptomatic. We propose an initial, small-scale pilot study to assess the feasibility of a larger trial to assess efficacy. The study aims to enrol 20 patients with severe lung disease, during a hospital admission. On top of their routine 6-minute walking test, patients will be asked to have a repeated test on HFNT and to fill in a questionnaire on their physical activity. As exploratory outcomes, we will measure distance walked during the tests, oxygen and carbon dioxide levels, breathing rate, and time to recover to baseline. Patients will be asked to rate their comfort and their breathlessness.

Aztreonam for Inhalation Solution (AZLI) for the Treatment of Exacerbations of Cystic Fibrosis. An Randomised, Crossover Pilot Study of AZLI Plus Intravenous Colistin® Versus Standard Dual Intravenous Therapy

This study aims to investigate whether it is safe and effective to use one inhaled antibiotic and one via a drip, instead of the current standard of practice of two antibiotics via a drip. Participants will receive the two different treatments the next two occasions they need to be admitted to Liverpool Heart & Chest Hospital with a chest infection. On one admission they will receive two IV antibiotics, on the other admission they will receive one IV antibiotic and inhaled aztreonam (known as Cayston®). The order they receive each treatment will be picked at random. Participants will have measurements taken including lung function tests, blood tests, CF quality of life questionnaires, and sputum samples will be sent to the University of Liverpool for advanced analysis of the effect each treatment has on bugs in the lung. It is hoped that this study may help provide more information toward a much needed expansion of therapeutic options available in CF.