Mr John Turgoose J.Turgoose@hull.ac.uk
Information Systems Manager - Hull Health Trials Unit
Mr John Turgoose J.Turgoose@hull.ac.uk
Information Systems Manager - Hull Health Trials Unit
Ms Bronwen Williams
Dr Chao Huang C.Huang@hull.ac.uk
Reader in Statistics
Professor Judith Cohen J.Cohen@hull.ac.uk
Director, Hull Health Trials Unit
Osteogenesis imperfecta (OI) causes skeletal fragility, bone pain, sarcopenia and reduced connective tissue resilience, shown as hypermobility and aortic root dilatation (in adults). There is no effective treatment for OI. Current agents only target skeletal tissue. Bisphosphonates (anti-resorptive) have reduced fracture frequency in mildly affected children; benefits are equivocal in severe cases. Adults have had no clear benefit of either anti-resorptive or anabolic therapy, e.g. PTH. Preclinical studies in severely affected OI mice show increased TGFβ signalling and increased bone turnover; anti-TGFβ antibody reduces bone resorption (by ~25%) and restores skeletal architecture. Inhibition of the TGFβ signalling pathway in other preclinical models where TGFβ signalling is increased (Marfan's, Duchenne Muscular Dystrophy) show improved skeletal and cardiac muscle outcomes. Inhibition of this pathway thus has the potential to improve both bone and muscle outcomes. We propose a novel approach, repurposing an existing drug (losartan) that reduces circulating TGFβ and is licensed for use in children and adults, to target both muscle and bone using a Bayesian approach to define an optimal biologic dose in 50 adults with OI aged 18-65. Additional efficacy outcomes - grip strength, bone microarchitecture by high resolution peripheral quantitative CT and OI-specific quality of life - will provide the basis for an application for Orphan Drug Designation and health economic evaluation.
Project Acronym | REMEDI4ALL |
---|---|
Status | Project Live |
Value | £922,056.00 |
Project Dates | Sep 1, 2022 - Aug 31, 2027 |
BREATHE: Breathlessness RElief AT HomE Apr 1, 2019 - Oct 31, 2021
A feasibility study to address this RQ:
Does a paramedic-administered short non-pharmacological complex breathlessness intervention improve breathlessness and reduce conveyance to ED for people with breathlessness crisis compared with usual practice...
Read More about BREATHE: Breathlessness RElief AT HomE.
LIPS: A prospective registry-based cohort study to monitor the diagnosis and management of acute leukaemia in pregnancy Jun 1, 2018 - Feb 28, 2022
Acute leukaemia (AL) is an aggressive but potentially curable cancer that can affect women of childbearing age. When a pregnancy is complicated by a diagnosis of AL, clinicians face a complex dilemma: to balance risking the mother’s survival through...
Read More about LIPS: A prospective registry-based cohort study to monitor the diagnosis and management of acute leukaemia in pregnancy.
PUMA: Paediatric early warning system (PEWS): Utilisation and Mortality Avoidance. A prospective, mixed methods, before and after study identifying the evidence base for the core components of an effective PEWS and the development of an implementation package for implementation and use in the UK Oct 1, 2017 - Oct 31, 2018
In 2011 a research study compared the child health outcomes and death rate in the UK with other European countries. It was worrying that UK measures of child health were amongst the worst in Europe. It is not clear why that is and further work needs...
Read More about PUMA: Paediatric early warning system (PEWS): Utilisation and Mortality Avoidance. A prospective, mixed methods, before and after study identifying the evidence base for the core components of an effective PEWS and the development of an implementation package for implementation and use in the UK.
Organic Disease masquerading as IBS: an RCT in primary care: The Lincolnshire Poacher Study (Promoting Optimal Assessment to Change Health and Engineer an Economic Revolution Jun 1, 2020 - Apr 30, 2023
Irritable bowel syndrome (IBS) affects up to 1 in 5 people in Britain. IBS can have a huge impact on a sufferer’s daily activities, general wellbeing, and overall health. IBS often carries major financial implications because of time lost from work,...
Read More about Organic Disease masquerading as IBS: an RCT in primary care: The Lincolnshire Poacher Study (Promoting Optimal Assessment to Change Health and Engineer an Economic Revolution.
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