Clinical Research Trials Research Team
Dr Monique Ryan (Head, Clinical Research Trials Research Team), Sian Rudge (Clinical Trials Coordinator), Dr Helen Young (Masters student), Dr Simon Grew (Masters student), Kristy Rose (Physiotherapist), Paula Bray (Occupational therapist), Dr Belinda Barton (Clinical psychologist)
The Clinical Trials research program of the INMR is aimed at identification of new treatments for inherited and acquired nerve and muscle disorders of childhood.
Clinical trials are research studies conducted with people who volunteer to take part. Each study answers specific scientific questions and tries to find better ways to prevent, screen for, diagnose, or treat a disease. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people and ways to improve health.
There are a number of different types of clinical trials:
- Interventional trials determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments.
- Observational trials address health issues in large groups of people or populations in natural settings.
- Prevention trials study ways to reduce the risk, or chance, of developing diseases.
- Screening trials study ways of early detection of diseases prior to the onset of any symptoms caused by the disease.
- Diagnostic trials study tests or procedures that could be used to identify diseases earlier and more accurately.
- Treatment trials are conducted with people known to have specific conditions, in order to answer specific questions about, and evaluate the effectiveness of, a new treatment or a new way of using a standard treatment. These trials may test many types of treatments, such as new medicines, vaccines, new approaches to physiotherapy or occupational therapy, or new surgical treatments.
- Supportive care or quality of life studies explore ways to improve the comfort and quality of life of patients and their families.
- Genetic studies may be part of one of the above studies or may be undertaken separately. These studies aim at identifying the genetic basis for inherited disorders.
At the INMR, we are currently undertaking a number of clinical trials aimed at improved understanding of a variety of nerve and muscle disorders of childhood. These studies include a study of clinical manifestations of Charcot-Marie-Tooth disease in early childhood, a study of early-onset inherited axonal neuropathies, and a study on the progression of muscle weakness in Duchenne muscular dystrophy.
We are also undertaking a number of trials of new treatments for childhood neuromuscular disorders. These studies are being undertaken at CHW singly or in collaboration with internationally-based clinical trials groups including the Cooperative International Neuromuscular Research Group (CINRG), which is based in Washington DC, and the European Neuromuscular Centre neuromuscular research consortium. Studies which have been recently completed include a study of the benefits of non-invasvie ventilation in children with respiratory failure related to neuromuscular disorders, and a multicentre study of the outcome of Guillain-Barre disease in childhood. We are now studying a number of new steroid and other treatments for Duchenne muscular dystrophy.
Selected Publications
Yates K, Gillis J, Festa M, North KN. Outcome of children with neuromuscular disease admitted to Paediatric Intensive Care. Archives of Diseases of Childhood. 2004, 89(2): 170-75.
This document was published on Tuesday, 9 August 2005.
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