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A Phase II dose escalation study of cyclophosphamide in haematopoietic stem cell transplantation in severe systemic sclerosis patients unfit for standard dose cyclophosphamide.
Patients with severe systemic sclerosis with cardiac complications requiring a stem cell transplant after their disorder progressing after standard treatment not deemed suitable for standard dose cyclophosphamide can be enrolled into an available cohort group to receive one of 3 different LOW doses of cyclophosphamide This study aims to reduce Treatment related side effects by reducing the intensity of conditioning, in particular cyclophosphamide dosage, in order to offer the potential benefits of stem cell transplant to patients normally excluded from HSCT whilst potentially maintaining efficacy. It is hypothesised that a lower dose cyclophosphamide stem cell transplant regimen of 12.5-35mg/kg/day in 3 cohorts will be tolerated with less toxicity than an historical cohort of 50mg/kg/day in patients with severe systemic sclerosis
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Orthotopic heart transplantation followed by autologous stem cell transplantation in patients with cardiac AL amyloidosis - a Phase II study
This study aims to investigate the safety and efficacy of autologous stem cell transplantation in AL amyloid patients with advanced cardiac disease. after a orthotopic heart transplantation. Who is it for? You may be eligible to join this study if you are aged between 18-65 years and have been diagnosed with cardiac AL amyloidosis. Study details All participants in this study are required to have previously received chemotherapy and a orthotopic heart transplantation before being enrolled in the study to received an autologous stem cell transplantation. Patients will undergo autologous stem cell transplantation (ASCT) within 3-6 months after OHT. patient will have an Autologous stem cell transplant using Melphalan 200mg/m2 on day -1 with stem cell collected given on day 0 previously from the patient before the study. All participants will be followed up every 3 to 6 months for a period of 5 years, in order to assess survival, and safety and efficacy of treatment. This pilot study will determine if treating patient with a stem cell transplant with cardiac amyloid after receiving a heart transplant will increase disease free survival
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Effects of statins on cognition in older adults with dementia
Background: Statins are effective in treating dyslipidaemia in older adults, reducing the cardiovascular related morbidity – however their adverse effects are more common and harmful amongst older adults. Additionally, the impact on cognition is unclear, with previous studies producing mixed results. The N-of-1 method has been demonstrated to be a feasible method of conducting deprescribing trials, generating strong-patient specific evidence as to the effects of discontinuing statin use on cognition. Aims: 1. To determine the effect on cognition of discontinuation and rechallenge with statins. 2. To determine the effects on quality of life and functional status of discontinuation and rechallenge with statins. Methods: A pilot interventional study of 30 older adults 80 years of age or older admitted to Royal North Shore Hospital, Sydney with current dementia diagnosis, and taking statins for at least 6 months will be conducted. Participants will be randomly assigned to statin and placebo treatment pairs with discontinuation and rechallenge of statins over the course of 4-months. At baseline (0-weeks), recruited patients will be subsequently randomised into either continuation of active statin treatment or placebo replacement for a period of 5-weeks. Participants will be followed at 5-weeks (Visit 2), 10-weeks (Visit 3) and 15-weeks (Visit 4). At each visit, patients will be crossed over into opposing intervention, either continuation of active statin or placebo. The primary outcome will be measured using the cognitive portion of the Alzheimer’s Disease Assessment Score (ADAS-CoG), which assesses the level of cognitive impairment on a 30-point scale with a psychometric test. The patients’ general practitioners will be contacted two months after trial completion to ascertain whether patients had discontinued, lowered dose, or continued their statin medication. Hypothesis: We hypothesise that patients will have improved cognition and quality of life when on placebo compared to when on statins.
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Does nifedipine improve implantation and pregnancy rates in women undergoing in-vitro fertilisation (IVF)/intracytoplasmic sperm injection (ICSI) cycles?
Implantation failure is the main factor affecting the success rate of IVF procedures. Excessive uterine contractions have been described as a potential reason for reduced implantation rates in IVF cycles. Contractile activity of the uterus could move the implanted embryo towards the Fallopian tubes or cervix/vagina or the embryo might even be expelled out of the uterus. Mechanical measures to reduce the uterine contractions at the time of embryo transfer include using a soft catheter without touching the fundus (the top of the uterus) and the use of ultrasound to guide embryo transfer. Various medications have been investigated to reduce uterine contractions, including cyclo-oxygenase inhibitors, B2-adrenoreceptor agonists, phosphodiesterase inhibitors. These have yielded variable results with atosiban, an oxytocin/vasopressin receptor antagonist, looking most promising for improving implantation and pregnancy rates. Calcium channel blockers are non-specific smooth muscle relaxants, used for the treatment of high blood pressure in adults and have an established role in preterm labour due to its uterine relaxing properties. In the uterus, the calcium channel blockers exert their “anti-contraction” effect by preventing the influx of extracellular calcium ions into the myometrial cell (the middle wall of the uterus) and have been demonstrated in laboratory experiments to have potent relaxant effect on human myometrium (the middle wall of the uterus). The most widely used and studied calcium channel blocker is nifedipine and there is evidence that this can be safely used in pregnancy. The objective of this study is to evaluate the effectiveness of nifedipine administration in improving implantation and pregnancy rates in IVF/ICSI fresh or frozen embryo transfers.
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Quality of life (QoL) in patients on Abilify Maintena in the private setting.
To date there has been no documented research on patients cared for in a depot clinic of a private setting (i.e. outside the public mental health sysytem) in Australia. This pilot study aims to evaluate the feasibility of conducting a RCT on the Quality of Life (QoL) amongst schizophrenia patients receiving Abilify Maintena and other anti-psychotics, as managed in a Private Depot Clinic. This study aims to evaluate the benefits of Abilify Maintena in the private specialist setting, independent of the public health service. This will ultimately validate the hypothesis that it can improve Quality of Life of patients, and achieve significant cost savings for the public services, thereby validating increased use of Abilify Maintena in the community.
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Trial to evaluate feasibility and benefits of adding Nasal High Flow in a hospital in the home setting following acute hospital presentations for exacerbation of chronic obstructive pulmonary disease.
Prospective feasibility trial with historic controls to explore the implementation and effectiveness of NHF in hospitalised COPD patients to facilitate discharge and 30 days of ongoing usage following discharge in the HITH setting to reduce 30 and 60 day readmission rate.
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Predictive Value of Bedside Lung Ultrasound in Preterm Infants with Respiratory Distress and the Evolution of Bronchopulmonary Dysplasia
Lung ultrasound is widely used within adult critical care with international evidence based recommendations for its utility in the assessment of patients with respiratory compromise. Further research is required to validate its clinical application within neonatal intensive care. Lung ultrasound is an attractive bedside clinical tool that is simple, non-invasive, quick to perform and well tolerated by even the most preterm of babies, using ultrasound equipment already in routine use. Bronchopulmonary dysplasia (BPD) is a process that starts soon after birth in preterm infants, with scarring and inflammatory changes within the lungs that may be slow to appear on conventional chest x-ray. Lung ultrasound performed in the first week of life may be more sensitive in recognising these changes earlier with the potential to assist in prediction of BPD and better direct therapeutic interventions (Raimondi et al 2014). A prospective observational cohort study will be undertaken performing lung ultrasound in 100 preterm infants born <28 weeks’ gestation admitted to KEMH neonatal intensive care unit receiving respiratory support. The study aims to determine if lung ultrasound can predict the development of BPD and respiratory outcomes.
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“Quercetin as an augmentation agent in Treatment Resistant Schizophrenia”
The hypothesis for this study is that a proportion of patients with schizophrenia may not respond to medication because the medication does not cross the blood brain barrier well enough. The blood brain barrier keeps substances out of the brain and is made up of several parts. One part is a group of proteins called “efflux pumps” or “transporters” which act to pump foreign substances out of the brain, back into the blood stream. We believe that some patients who do not respond to medications for schizophrenia have such fast and efficient efflux pumps that the medications cannot get into the brain effectively. The speed and efficiency of these efflux pumps are determined by the genes for these pumps. The particular pumps that are of interest in this study are called P-glycoprotein (PgP) and Breast Cancer Resistance Protein (BCRP). This study will test the addition of a natural product, quercetin, to the patient’s regular medicine. Quercetin is known to slow down these pumps. The aim of the study is: To show that quercetin has beneficial effects when added to the usual medications used in in schizophrenia Objectives: -To figure out the how quercetin has a beneficial effect. -To identify the symptoms of schizophrenia which responded best to quercetin use -To see if there are groups of genes that indicate fast efflux pumps in patients with schizophrenia who do not improve with the usual medications.
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A double blind, placebo controlled, randomized crossover, pilot study of topical RM191A spray for the relief of neuropathic pain
This is a double blind, placebo controlled, randomized crossover, pilot study assessing the therapeutic effects of RM191A in participants suffering from neuropathic pain. Each participant receives both treatment (separated by three days of washout period) and randomisation is used to determine the order in which the participant received each treatment. The 10% RM191A spray will be applied four times a day at 8AM, 12PM, 4PM and 8PM with up to eight sprays per area. Changes in intensity of neuropathic pain will be assessed by NPRS.
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Can an increase in consumption of dietary fibre improve gut flora profile, bowel habit and sense of wellbeing in healthy adults?
This project aims to discover new understanding regarding whether an increase in dietary fibre will improve the profile of gut flora (i.e., the micro-organisms that aid digestion), bowel habit and sense of wellbeing. There is strong evidence suggesting dietary fibre is essential for good health. This project aims to understand better the fibre-health relationship.