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Nemaline myopathy (NM) is a form of genetic muscle disease characterised by the presence of numerous abnormal dense protein inclusions (nemaline bodies) on muscle biopsy. Children NM have varying degrees of muscle weakness with reduced functional capacity and exercise tolerance. Due to the strong association between muscle and bone (the muscle-bone unit) children with NM are also a risk of disuse osteopenia and minimal trauma fracture. To date, the optimal exercise regimen to increase muscle strength (force and power) is uncertain in NM. A training method that maintains muscle function throughout childhood and into adult life has the potential to improve the quality of life of children with NM. Whole body vibration training (WBVT) using a vibration platform is an emerging, simple and safe training method, which has been shown to improve muscle function and fitness in adults and children. In addition, WBVT has been shown to improve osteopenia in various populations. This project aims to evaluate the effect of 24 weeks WBVT on muscle and bone parameters in children with NM. Home-based Exercise Program for Children with Nemaline Myopathy: Ten children with nemaline myopathy, 4 – 18 years old will be enrolled into this study. The children will undertake a home-based WBVT program using the Galileo (trademark) vibration platform7 days a week for a 24 week (six-month) period. The WBVT period will be preceded by a 6-month observation period to establish the natural history of muscle function and bone density without intervention in each child. A series of investigations at baseline, the end of 6 months of observation and after 6 months of WBVT will evaluate at the effects of WBVT on muscle strength (force, power and efficiency), muscle size, functional mobility, bone strength and quality of life.

The purpose of this study is to explore the importance of gut sweet taste sensors in the regulation of sugar control, appetite and body weight during health, obesity and after bariatric surgery. The findings of the current study may provide further insights into the mechanisms underlying the development of obesity and diabetes mellitus, which, in turn, can have a great potential therapeutic implication. Hypothesis: We hypothesise that the number of these sweet taste receptors will be markedly reduced in the small intestine of morbidly obese patients but will be increased after bariatric surgery, leading to increased incretin responses and subsequently, better glucose control and body weight (via high GLP-1). We further hypothesise that the greater release of GLP- 1 (thus, release of insulin) after RYGB over LAGB is related to the higher expression of small intestinal sweet taste receptors, leading to better glucose control and weight loss in these patients. This study, therefore, aims to evaluate the expression of small intestinal sweet taste receptors before and after a sugar ‘meal’ and its relationship to GLP-1 concentration, glucose control and body weight in morbidly obese subjects and patients who undergo RYGB and LAGB.

A multi centre, investigator blind, study in a total of 45 subjects with mild to moderate acne vulgaris aiming to evaluate the efficacy, safety and tolerability of topical tretinoin formulated with TPM compared with a commercially available tretinoin formulation, and a vehicle.

Age-related macular degeneration (AMD) is a major cause of visual impairment in advanced countries, responsible for nearly half of all legal blindness in Australia. Due to increased life expectancy, the number of people with this progressive late onset disease will double by 2025. Aspirin could prevent or delay the onset of AMD in older persons but its bleeding risk also needs to be considered. This ASPREE-AMD project will determine whether treatment with low dose aspirin reduces incidence or progression of AMD. ASPREE-AMD study is a sub-study of the large-scale randomised controlled trial ASPREE, which is registered with the International Standardised Randomised Controlled Trials Register. Title: Aspirin in Reducing Events in the Elderly (ASPREE). Number: ISRCTN83772183.

Knee osteoarthritis (OA) is a major public health problem. It develops over many years, with progressive loss of articular cartilage. In people over 70, even without radiographic OA, most will have cartilage damage. Loss of cartilage is associated with pain and reduced function. Thus in an older population, it is important to reduce knee cartilage loss, and prevent structural progression. Low dose aspirin, used in the prevention of cardiovascular disease, may also affect cartilage by a variety of mechanisms. We have pilot data in 2 independent studies suggesting that low dose aspirin may reduce cartilage loss by more than 50%. The ASPirin in Reducing Events in the Elderly (ASPREE, ClinicalTrials.gov identifier NCT01038583, website www.aspree.org) study is a current 5 year randomised placebo controlled trial testing whether low dose aspirin in healthy adults > 70 years prevents cardiovascular disease, cancer and functional decline. Within this larger study (ASPREE parent study) we will examine in the ASPREE Knee Sub-study whether the use of low dose aspirin reduces change in knee cartilage volume in older adults.

This project is investigating minimum standards of practice and education for physiotherapists working in Australasian intensive care units. The lack of consistency in professional standards and dedicated training poses a substantial threat to the practice of physiotherapy and the credibility of the profession within the critical care unit. To provide 24 hour, 7 day a week services, many physiotherapy staff are required to contribute to critical care rosters, regardless of their primary discipline or interest (e.g. orthopaedics). Traditionally physiotherapists complete a tailored, in-house intensive care orientation including theory, clinical skills training and clinical practice until deemed competent by another staff physiotherapist. This process is subjective, requires extensive familiarization and additional skills in assessment and treatment. Educational requirements for competency training are not standardized across or within Australia or NZ, and are established by individual networks, with large variability in the content, requirements and duration of the education provided. A panel of participants will be selected to reflect the range of stakeholders who have an interest in the outcomes of the project (Boulkedid, Abdoul, Loustau, Sibony, & Alberti, 2011). Sixty-one participants from across Australia and NZ will be invited on to the panel, including Physiotherapy Specialists/Fellows in critical care, senior physiotherapy critical care clinicians, and physiotherapy academics in cardiorespiratory education. The Delphi survey plan will consist of two or three rounds of surveys.

This study aims to define the role of iron deficiency and iron deficiency anaemia in Paediatric Inflammatory Bowel Disease. Iron deficiency with or without anaemia is prevalent in inflammatory bowel disease (IBD) and an adverse impact on quality of life and cognitive function. These effects are likely to be equally if not more severe in the paediatric population. Correction of iron deficiency in IBD is difficult because of ongoing losses and because oral iron is poorly tolerated and bioavailability is impaired in states of inflammation. Given this, intravenous iron preparations are increasingly being used to correct iron deficiency. Previous preparations were complicated by a high rate of anaphylaxis and of iron toxicity which limited the doses administered leading to incomplete correction. We aim to show that the treatment of iron deficiency and iron deficiency anaemia in children using a newer, safer compound with no reported anaphylaxis nor iron toxicity, Ferric Carboxymaltose, is safe and effective in children and that treatment to correction of iron deficiency improves quality of life. This compound has been safely and effectively given, in corrective doses to over 10,000 patients aged 14 and above and there is no reason to indicate it is not equally safe and efficacious in younger children. We also aim to demonstrate improvements in cognitive function after correction of iron deficiency and that the administration of ferric carboxymaltose does not significantly increase markers of lipid peroxidation in paediatric inflammatory bowel disease. The patients included in this prospective open study will be children (six and older) treated for Inflammatory Bowel disease at the Royal Children's Hospital in Brisbane, with documented iron deficiency or iron deficiency anaemia (as defined by published guidelines). Baseline records of iron status (Ganzoni formula, FBC, IS) and quality of life will be obtained through blood tests and the IMPACT III QOL questionnaire (previously validated). A cognitive assessment will also be recorded by a qualified psychologist using a WISC-R. Treatment with Ferric Carboxymaltose will be undertaken (dose 15mg/kg body weight up to 500mg as per current hospital infusion guidelines for patients aged 14 and over. Following this, repeat iron status and quality of life information will be obtained at 8 weeks, with a repeat assessment of cognitive function. Primary endpoints will be defined as correction of total iron stores and improvement in cognitive function and QOL measures.

Atrial fibrillation (AF) is the most common cardiac arrhythmia affecting one in four people in the adult population. AF is a major cause of admission to hospital and represents a major public health burden. AF is an independent risk factor for stroke, with a near five fold excess of stroke observed in patients with AF. It is of even greater concern that patients living with AF experience a range of physical and psychological symptoms which are distressing and impact negatively on their quality of life. In addition, those with AF have a high prevalence of known cardiovascular risk factors such as being physically inactive, overweight and having hypertension. Despite the evidence that cardiovascular risk factors are poorly controlled in patients with AF, they are typically not included in risk factor reduction programmes. Therefore, in this study, we aim to determine if a simple patient-centred, home-based risk factor management program can improve cardiovascular risk and quality of life in patients with clinically diagnosed AF. The study will be a pilot trial using a pre-post design with 3 month follow-up of clinical outcomes involving 20 people with AF. A mixed methods design comprising both quantitative and qualitative methods will be utilised. Study volunteers will have a baseline assessment and an initial in-person module selection and goal-setting session of approximately one hour. All participants will take part in a 3-month patient-centred intervention with tailored risk factor management and an individually tailored community-based exercise program. This program is based on health coaching and motivational interviewing principles and the extensive experience the researchers have with modular secondary prevention of cardiovascular disease and behaviour modification in high risk patients. Simultaneous, ongoing consultation with their general practitioner and specialist physician will be encouraged as part of standard medical care. Outcome assessments for all participants in the pilot study will be made at three months after baseline assessment.

There is a need to better understand the mechanisms involved in peritoneal membrane deterioration in patients undergoing peritoneal dialysis. We have found that a recently discovered particle (calciprotein particle, CPP) is present at high levels in drained out peritoneal dialysis fluid (PDF). Experimental work suggests that CPP may promote inflammation and calcification in certain situations. This is a pilot study to ascertain whether differences in PDF composition, in particular with respect to fluid glucose and calcium concentration, effect the formation and levels of CPP present in drained PDF. We hypothesise that PDF containing higher glucose and calcium concentrations would be associated with greater intra-peritoneal CPP accumulation, which may predispose to peritoneal inflammation, calcification and ultimately, technique failure.

Our 3-month randomised placebo controlled double-blind trial study aims to investigate the effect of aged garlic extract on blood pressure and other cardiovascular biomarkers including cholesterol, homocysteine and arterial stiffness in a group of adults with uncontrolled hypertension.