This is a multicentre, prospective, non-interventional observational cohort study of patients who are planned to receive, or already receiving Epidyolex under real-world conditions of clinical care. This study is designed to address a Pharmacovigilance Risk Assessment Committee (PRAC) request to conduct a Category 3 Post-Authorisation Safety Study (PASS), as requested in the Risk Management Plan (RMP) at the time of approval. While 12 safety concerns were noted in the RMP, PRAC emphasis was on the need to further characterize the ...

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This study will evaluate the expression of neurofilaments and of other relevant molecules in blood samples taken from individuals with amyotrophic lateral sclerosis at different time points during the development of the disease. Our aim is to validate easily accessible disease biomarkers functioning as reliable predictors of disease severity and capable of providing information about the stratification of the disease phenotypes. Control groups will include healthy individuals and patients with a compressive radiculopathy. Recruitment will take place in 4 motor ...

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The study hypothesis is that a strategy of universal primary prevention of anaemia during pregnancy with oral iron supplementation will impact the composite primary outcome of pre-term birth,stillbirth,neonatal death and small for gestational age (SGA).

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RATiFY is a non-randomised,multicentre,phase II trial. Eighty patients will be recruited and,following initial treatment with 3 cycles of tislelizumab and PET-CT with ceCT scan,will be assigned to 1 of 5 treatment groups (Groups A to E) based on disease stage,risk,and response. Patients with early stage lymphoma showing good response (Group A) will receive radiotherapy and further tislelizumab for up to 2 years,but no chemotherapy. All other patients (Groups B-E) will receive further tislelizumab with AVD chemotherapy for between 2 and ...

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PEARLS is a 2-arm,parallel-group,individually randomised,open label assessor-blinded,multicentre,superiority trial with an internal pilot phase. It is a pragmatic trial and the trial participants in both intervention and comparator group will receive commonly prescribed topical corticosteroids (TCSs),which is tailored for the participant. The only difference between the treatment groups is the treatment regimen/strategy. There are number of TCS products available on the market and all available brands and forms can be prescribed to participants. Participants will be randomised to one of ...

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Every year more than 700,000 women give birth in the United Kingdom. Of these at least 8700 nearly die – called a “near-miss”, and 70 die. Many more women suffer harm, often with effects lasting for life. Women from less wealthy areas and particular ethnic groups are more likely to come to harm. "Vital signs" include measurements of blood pressure, heart and breathing rates. Doctors and midwives use tools that score vital signs to spot women becoming unwell. These tools are ...

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ADVANCE is a longitudinal cohort study supporting scientific research by members of the 'NASH Disease Translational Accelerator' (NASH-DTA) Consortium. The overarching aim is to leverage a greater understanding of disease pathophysiology to develop robust methods for diagnosis, risk stratification and therapy for patients with Non-Alcoholic Fatty Liver Disease related advanced liver fibrosis/cirrhosis (“compensated advanced chronic liver disease, cACLD”). To achieve this we will assemble a longitudinal observational cohort of well-characterised patients with Non-Alcoholic Fatty Liver Disease (NAFLD) related advanced liver ...

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This is a phase IIIa,multi-centre,randomised,doubled-blind,parallel-group,placebo-controlled trial,with a 104-week blinded treatment period followed by a 26-week open-label extension. The aim of the trial is to assess whether 1260mg of ambroxol daily can slow down the progression of Parkinson’s disease (PD),in comparison to a matching placebo,and to establish the safety of long-term use of ambroxol at this dose. 330 participants will be recruited from approximately 10 UK sites over a 24-month period. Data analysis will be performed by UCL CCTU’s statisticians at ...

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Reflux disease can severely impact upon quality-of-life and lead to complications, including ulceration of the oesophagus. It is often controlled with self-help measures and medication. However, sometimes surgery is recommended. The current standard surgical treatment is called a fundoplication. This operation is carried out through keyhole (laparoscopic) surgery, and tightens the lower oesophagus to prevent reflux. Fundoplication is very safe and improves the quality-of-life of most patients. However, many patients have gas bloating, difficulty swallowing and recurrence of their reflux ...

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Tuberculosis (TB) kills more people than any other single infectious disease. We really need an effective vaccine to prevent people getting TB, but we don’t understand what sort of immune response is needed to protect people. The very early responses in TB infection, is not well understood, partly because it is difficult to study as most of the changes happen before people get symptoms. This study has been designed to help to answer two key questions about the early immune ...

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