ANZCTR search results

Atypical hemolytic-uremic syndrome is a serious, life-threatening rare and chronic disease believed to be caused by genetic mutations. Current treatment for the disease is inadequate. Due to the uncontrolled complement activation seen in aHUS patients and the previously shown activity of eculizumab to selectively inhibit terminal complement activation, it has been decided to look in to the use of eculizumab in the treatment of serverely affected aHUS patients.

HCM is the most common cardiovascular genetic disorder and can cause significant morbidity and mortality, including the most serious complications of heart failure and sudden death. At present, there are no proven pharmacological therapies that either prevent or cause regression of clinical features. This is largely due to a lack of knowledge in our understanding of the molecular and functional consequences of the disease-causing gene mutations leading to the clinical disease. The proposed study will aim to test whether cardiac energetic compromise is a central pathophysiological mechanism in HCM. We will investigate potentially beneficial treatments based on our hypothesis, by trialling the use of metformin in an HCM group. If the results from this ‘proof of concept study’ are confirmatory, it could pave the way for larger multi-centre randomised studies (with longer duration of treatment) with either metformin or facilitators of fatty acid oxidation such as ranolazine. If realised, these treatments could prevent the vicious cycle of cardiac hypertrophy and myocardial dysfunction seen in hypertrophic cardiomyopathy, leading to improved morbidity and mortality. These investigations will establish early phenotype changes in HCM patients and provide insights into potential therapeutic interventions for a condition that currently has little therapeutic evidence base. This therapeutic sub-study is linked to the main study ACTRN12611000479909.

The purpose of this study is to compare the levels of discomfort a patient experiences, to compare the effects on blood components and to compare the surgical outcomes associated with different modes of bowel preparation before gynaecological laparoscopic surgery. Laparoscopic surgery is a minimally invasive surgical technique, for which you have already been admitted to this hospital. Prior to surgery, it is standard procedure in this department for patients to take mechanical bowel preparation. This involves following a diet and then taking a proprietary drink to assist in emptying the bowels. Traditionally, the emptying of the bowels is believed to improve the visualisation of abdominal contents as well as potentially decrease complication rates involving the bowel, though there is not a lot of scientific proof that this is the case. Recent research in gynaecological and bowel surgery has shown that mechanical bowel preparation may not be necessary. In this study, patients will be randomly assigned to one of three groups: 1. Women in group one will only fast (not have anything to eat or drink) before their surgery 2. Women in group two will fast and undertake a minimal residue diet 3. Women in group three will undertake a minimal residue diet and take mechanical bowel preparation as directed. You will have a 33% chance of being in any of the three groups. Neither the doctor nor the study participant can decide which bowel preparation the patient will undertake. This study will be a single blind trial. This means that the doctor will not know which bowel preparation the participant has taken. When you have been assigned to one group, we will provide a detailed outline of the bowel preparation you need to undertake. A study team member will contact you before your surgery to ensure that you know which group you are in and to remind you of the procedures. The knowledge gained from this study will allow us to estimate whether one type of bowel preparation is significantly better for patients, enabling us to provide a better quality of care for all women.

The purpose of this study is to evaluate if using a checklist immediately after the resuscitation of severely injured patients in order to ensure that certain elements have not been forgotten improves care for those patients.

The first purpose of this study is to examine if comfortable in shoe foot orthoses improve the symptoms of anterior knee pain over a short-term period more than a control group who does not receive orthoses. The second purpose is to determine what physiological effect is causing any improvement. We hypothesise orthoses will improve symptoms more than a control and that these improvements are occuring via adaptations in the activity of the lower limb muscles.

Antenatal magnesium sulphate is recommended prior to preterm birth at less than 30 weeks’ gestation for neuroprotection of the fetus. Whether there are benefits at later gestations is uncertain. The primary aim of this randomised placebo controlled trial is to assess whether giving magnesium sulphate compared with placebo to women immediately prior to preterm birth between 30 and 34 weeks' gestation reduces the risks of death or cerebral palsy in their children at 2 years' corrected age. years' corrected age.

Parkinson’s disease primarily affects the way a person is able to move or walk and causes muscle stiffness and tremor, which can be very disabling. However, these patients also suffer from non-motor symptoms. These non-motor symptoms include depression, cognitive impairment, and sleep disturbances. As the disease progresses these non-motor symptoms have been shown to have a greater impact on the patients quality of life (QoL). Obsessive Compulsive Spectrum Disorders (OCSD) are a group of psychiatric disorders that have been demonstrated in patient with Parkinson’s disease. We hypothesized that OCSD have an impact on the QoL of patient’s with idiopathic disease. We aim evaluate the prevalence OCSD in patients with idiopathic Parkinson’s disease and its impact on the patient’s QoL.

The purpose of this study is to determine which of two different approaches in accessing an artery, transfemoral (via the artery in the groin) or transradial (via the artery in the wrist), both of these are currently used in clinical practice, is least associated with bleeding and procedural related complications. It will also determine whether using an ultrasound machine to guide the arterial access will reduce bleeding and other procedural related complications.

Thiazolidinediones (TZD) are a class of drug commonly prescribed to manage type 2 diabetes. While the exact mechanism of action is unknown, efficacy of the drug is closely correlated with improved circulating concentration of a protein, adiponectin, secreted by fat tissue. Adiponectin is decreased in many diseases states including type 2 diabetes. Adiponectin is secreted as multimers of different sizes, termed low molecular weight (LMW) and high molecular weight (HMW), and while an increase in circulating adiponectin is beneficial, it is more specifically an increase in the HMW multimers that is correlated with improvement in health. An improvement in adiponectin profile refers to an increase in the proportion of the multimers present as HMW and may also be concomitant with an increase in total adiponectin. Previous studies have demonstrated that high dose Vitamin C alone can improve glucose control in obese and diabetic patients. Researchers involved with the proposed research project have demonstrated that combining Vitamin C with TZD treatment has a synergistic effect on adiponectin profile in vitro in a human adipose cell line. Vitamin C alone improves adiponectin profile 1.7 fold, while improvements are 5 fold over baseline when in combination with TZD. Combining the evidence from previous studies and these in vitro observations demonstrates a potential for combining TZD with high dose Vitamin C to get an improved treatment for managing type 2 diabetes. Research Design and Methods: Patients will have a two week wash out period to control vitamin c levels, and then two weekly intervals on escalating doses of oral vitamin C for 8 weeks (4 doses, 500 mg / day, 1000 mg / day, 1500 mg / day and 2000 mg / day). Each patient will serve as their own control, with assessment of total and HMW adiponectin at the beginning and end of the trial and at dose escalation points in between as primary endpoints. Other indicators of improvement in metabolic parameters will also be monitored.

This study will investigate the effects of chronic administration of two natural supplements on cognition, cardiovascular function and biological measures, in comparison to a placebo. The primary aim of this project is to determine whether 12 months administration of the natural supplements Bacopa monniera (EBm) and Pycnogenol (PYC) improve cognitive functioning in a healthy older population. The secondary aims of this project are to investigate the mechanisms underpinning any cognitive effects of EBm and PYC by examining the relationship between cognition, biochemical, neuroimaging and cardiovascular measures and to investigate the time course of any improvements. The study will follow a double-blind, randomised, placebo-controlled, between-group design. Participants will be required to consume one of three types of supplements (EBm, PYC, or placebo) daily for 12-months. We will measure performance on different cognitive tasks, mood, brain imaging (for a sub-group of participants), gut microbiota (for a sub-group of participants), cardiovascular and biochemical measures before treatment (baseline), 3-months, 6-months and 12-months after consuming the treatment. In addition, as a sub-study to the main project 150 participants will be recruited to take part only in baseline assessments. The participants will undergo the same measures (excluding brain imaging) as for the main study, but will not receive any intervention and will not be required to attend follow-up assessments. This sub-study will allow further exploration of the relationship between GI microbiota and the other outcome measures including cognition, mood and cardiovascular function.