1507115 / ILD Genetics

Identification of disease susceptibility genes and autoantibodies associated with the development and clinical characteristics of interstitial lung disease (ILD) in patients with and without proven connective tissue diseases (CTDs). (Study due to close 01/03/2021)

1507118 / Early low dose steroids for adults admitted to hospital with influenza-like illness during a pandemic: a randomised placebo controlled trial

During a pandemic, influenza infection can be severe because the population has little or no immune protection to the new virus. In such circumstances, severe influenza may lead to hospitalisation, admission to intensive care or death. A pandemic also poses challenges for health care systems which may be overstretched leading to further detrimental impacts on patient care. Current treatment options for influenza, including antivirals, are only partly effective at reducing adverse outcomes. Clinical trials of corticosteroids in patients with pneumonia or severe ‘blood poisoning’ have reported improved outcomes. There is uncertainty whether corticosteroids might also improve the recovery of patients with severe influenza infection. The ASAP trial will investigate whether low-dose corticosteroids (dexamethasone 6 mg once a day for 5 days) given to adults (aged 16 years and over) within 24 hours of admission to hospital with an influenza-like illness in addition to standard treatment are beneficial.

The influenza pandemics of the last century have typically spread in 2 or more waves, each wave approximately of 6 weeks duration. Therefore, we aim to complete recruitment (2200 patients) within a 6 week period corresponding to the first pandemic wave in order to inform pandemic management before the subsequent waves strike. (Study due to close 01/05/2025)

1510142 / EME-TIPAC

Pulmonary fibrosis is a condition with limited treatment options. In a previous study we showed that people with pulmonary fibrosis who regularly took an antibiotic (called cotrimoxazole) were 5 times more likely to be alive after one year than those that didn’t. However, treatments for pulmonary fibrosis have now changed and we are trying to find out if cotrimoxazole is still as effective. We want to know whether cotrimoxazole, when given alongside current treatments, improves life expectancy and/or reduces the chances of being admitted to hospital for people with pulmonary fibrosis and, by measuring biomarkers (see below, how it may be working. We are aiming to recruit 330 participants from different regions throughout the UK.

All patients must have some degree of breathlessness and reduced breathing tests and not have major health problems, problems with their liver or kidneys. After completing initial assessments and safety blood tests, participants will be randomised to receive either cotrimoxazole 960mg twice daily or a placebo tablet for between 1 year and 3.5 years depending on when they start the study. We will record the time until either a participant is admitted to hospital, has a lung transplant or dies. Questionnaires, breathing tests and blood for biomarkers (chemicals that allow us to understand about pulmonary fibrosis) will be completed or taken after 3 months, 6 months then every 6 months until the study ends.

Patients will be invited to provide a blood sample for genetic testing. Fifty patients will be invited to have a bronchoscopy (lung camera test) to find out if cotrimoxazole has an effect on the numbers or amount of inflammatory cells, biomarkers and bacteria (using traditional and new techniques). It is hoped that this study will confirm whether cotrimoxazole has a benefit for patients and, if so, how it may be working. (Study due to close 31/05/2018)

1611335 / A randomized, double blind controlled trial comparing rituximab against intravenous cyclophosphamide in connective tissue disease associated interstitial lung disease

Interstitial lung disease (ILD), which is characterized by inflammation and scarring of the lungs, is the leading cause of death in systemic sclerosis (SSc), and a major cause of morbidity in many other connective tissue diseases (CTDs) a group of conditions that are caused by over activity of the immune system. If connective tissue disease associated interstitial lung disease (CTD-ILD) is severe or progressive, immunosuppressive treatment (treatment used to damp down the immune system), such as intravenous cyclophosphamide, is required to suppress inflammation and minimise progressive lung scarring.

Occasionally, even intensive standard immunosuppressive drugs fail to control lung inflammation, and progressive lung damage may develop that ultimately results in death. Rituximab, a novel immunosuppressive therapy, has been proven to be of benefit in suppressing inflammation associated with immune system over activity, including pulmonary inflammation in CTDs.

In a randomised, controlled, double blind study, we will compare the effectiveness of rituximab against cyclophosphamide as first line therapy in patients with severe, progressive CTD-ILD. (Study due to close 31/08/2019)

1801561 / Quality of life measurement in people with cystic fibrosis registered with the UK CF Registry

So far to our knowledge, no large scale, prospective study has been conducted worldwide to assess the quality of life in cystic fibrosis population that contains a sufficient sample size and a range of population characteristics to inform longitudinal observation, risk prediction models and economic evaluation parameters.

In order to be able to assess all the aforementioned parameters, we will be inviting people with cystic fibrosis to participate to a suite of online questionnaires of Quality of Life measurement. We propose the application of an online based questionnaire that will entail 3 different already validated instruments to capture all the relevant parameters of CF patients quality of life and utilization aspects.

The Cystic Fibrosis Quality of Life (CFQoL) questionnaire to measure the CF specific quality of life of adult and adolescent patients, the EQ-5D tool which is a generic tool with limited clinical utility but it is a recognised measure for use in cost benefit analyses and finally the personal well-being questionnaire of the Annual Population Survey (APS) conducted by ONS. These questionnaires will be completed by patients that are registered with the CF Registry and linked to their demographic and annual encounter clinical data. The CF Registry is sponsored and managed by the UK Charity, the Cystic Fibrosis Trust and is a research database holding data of people with CF in the UK.

About 99% of all people with CF in the UK have provided explicit consent to the Registry. Patients will be invited to participate through Patient Information Sheets that will be sent along with their annual review letters once a year. On the day of their annual review they will be given a unique code along with a website address to securely log in and complete the questionnaires. (Study due to close 01/09/2019)

1802593 / A randomized, double-blind, placebo-controlled phase II study
to evaluate the efficacy and safety of spx-101 inhalation solution in subjects with cystic fibrosis (hope-1 study: hydration for optimal pulmonary effectiveness)

Evaluation of potential mucus clearing treatment for all patients with CF regardless of mutation (Study due to close 10/11/2018)

1804573 / A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Safety, Tolerability and Efficacy of Pirfenidone in Patients with Rheumatoid Arthritis Interstitial Lung Disease

A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of Safety, Tolerability and Efficacy of Pirfenidone in Patients with Rheumatoid Arthritis Interstitial Lung Disease  (Study due to close 31/10/2019)