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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This is a FTiH modular, Phase I/II, open-label, multicentre, dose escalation and expansion study in participants with r/r haematologic malignancies. The study consists of several study modules, each evaluating the safety, tolerability, PK, pharmacodynamics, and efficacy of orally administered AZD3470 as monotherapy and in combination with other anticancer agents. This study will follow a modular protocol design evaluating AZD3470 as monotherapy and in combination with other anticancer agents. New cohorts (including further monotherapy expansion) and new modules for combination treatments may be ...

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Childhood and adolescent cancers comprise a heterogeneous group of rare diseases, mostly distinct to those diagnosed in adults. Survival of children and adolescents with cancer has considerably improved in recent decades, but cancer remains the most common cause of disease-related mortality in developed countries. In the case of disease relapse, the prognosis for paediatric patients is poor with survival rates of < 20%. Better understanding of tumor biology has led to exponentially increased development of novel targeted therapies and immunotherapies in ...

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Nemaline myopathies(NM) are genetically determined conditions that usually present with muscle weakness, breathing problems and difficultly feeding. The severity of NM varies from significant muscle weakness requiring a wheelchair, breathing and feeding support with signifcantly reduced life expectancy to milder forms in which the person remains able to walk and has a normal lifespan. Current treatments for people living with NM are supportive only. Several potential therapies are in development which may be available in the next 5-10 years. ...

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Pancreas transplantation is an effective treatment for patients with severe complications of diabetes. It improves quality-of-life and life-expectancy by returning blood sugar levels to normal and removing the need for insulin. Most pancreas transplants are performed at the same time as a kidney transplant for patients with kidney failure due to diabetes. When a donor pancreas is removed from the body it is stored in specialist fluid in an ice box. This method slows down cell activity reducing, but not ...

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People with familial hypercholesterolaemia (FH) have raised cholesterol levels in their blood from birth. Cholesterol is a type of fat our bodies need but having raised levels over a long time causes heart disease, such as heart attacks. To prevent future heart disease, guidelines recommend starting cholesterol-reducing medication, usually statins, by age 10. However, there remain unanswered questions about what is the right age and cholesterol level at which to start statins to prevent later life heart disease. Considerations are: avoiding ...

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This multicenter, multinational, natural history study is designed to retrospectively and prospectively collect clinical data in participants with genetically confirmed DOK7-CMS. No IMP will be administered. Participants will attend up to 3 study visits and complete home digital physical activity monitoring for 1 week after each visit. The assessments will evaluate participants’ symptoms and quality of life to understand disease activity in patients with DOK7-CMS better and may inform future study design. Approximately 30 male and female participants ≥18 years of age ...

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Multisite,multi-arm,open-label,pragmatic randomised controlled trial with integral internal pilot and concurrent economic evaluation. The trial is predicated on two primary comparisons of (1) a monotherapy (i.e. Iron) and (2) a combination therapy (i.e. Iron + EPO),compared with a single common control group (usual care) respectively,in a superiority hypothesis testing framework.

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