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Lymphangioleiomyomatosis (LAM) is a rare lung disease restricted to adult women. This may occur either sporadically (s-LAM) or in association with tuberous sclerosis (TSC-LAM). The lungs of patients with LAM are infiltrated by smooth muscle cells (LAM cells).There is a very limited evidence base for treatment of LAM and it is only recently that the first randomised trials have been initiated. The LAM clinic at St Vincent’s follows the largest number of women with LAM in Australia, currently approximately 40 patients. The first ever trial into the efficacy of doxycycline in LAM was completed at St Vincent’s LAM clinic, and this new study aims to assist the breathlessness associated with this disease and evaluate the mechanisms underlying this. Indacaterol is a novel, inhaled, once-daily, ultra-long-acting beta 2-agonist for the treatment of chronic obstructive pulmonary disease (COPD). Bronchodilator agents are frequently prescribed in LAM but have never been the subject of a clinical trial. Long acting beta2-agonists have been described as having anti-proliferative effects on smooth muscle in vitro as well as in vitro, but these agents have not yet been investigated in LAM. It is theoretically possible that women with LAM will respond clinically, and it is also possible that indacaterol might have a beneficial effect within the airway. This trial will evaluate indacaterol in LAM using a classical study design ie. a double blind placebo controlled trial, in 15 women with LAM.

The Tailored EDV study (ENG4) is an open-label feasibility study of a single delivery agent VEDVsPayload containing clinician chosen therapeutic payload(s) (cytotoxic drug, siRNA or miRNA). Who may be eligible? Participants must have histological or cytological confirmed advanced solid tumours, who have no curative treatment options. Eligibility also requires participants to have an archived tumour sample(s), or recent primary or metastatic tumour biopsies, (paraffin block or slide) for analysis of Epidermal Growth Factor Receptor (EGFR) expression. In addition, radiological assessment by MRI, CT or PET scan confirming measurable disease is required for study entry. Study details: Participants will receive VEDVsPayload once a week for 8 weeks, via a 20 minute intravenous infusion. This is known as 1 treatment cycle. There are four potential cytotoxic chemotherapy drugs, in addition to potent anti-tumour siRNA or miRNA, that can be individually selected as the VEDV payload. The VEDV can be packaged with a single or multiple payload, dependent on the pre-identified sensitivity of a patients cancer cells to one or more of the cytotoxic agents, so uniquely targeting the individual’s disease. The investigational treatment is given as an out-patient. Participants will be monitored closely for any adverse effects by the collection of regular blood tests to monitor haematological and liver function, in addition to physical examinations and monitoring of vital signs (including resting pulse, respirations, blood pressure and temperature). Participants will be required to spend between 4 and 6 hours in the hospital at each visit. Tumour evaluation using the same method of assessment as at screening, MRI, CT or PET scan, will be performed every 9 weeks (after each 8 week treatment cycle). If the patient does not fulfill any of the criteria for withdrawal they may continue to receive further cycles of VEDVsPayload for up to a maximum treatment period of 12 months. If new in-vitro data emerges from the testing of patient’s tumour samples collected either from biopsy or from circulating tumour cells, then this data will be regularly reviewed by clinical treating team and the chief scientists at EnGeneIC Pty Limited. If the data suggests that the patient may derive increased clinical benefit by combining or changing the therapeutic payload packaged in VEDVs, then the patient may receive treatment with a different VEDVsPayload. Follow-up: A safety follow-up visit will be performed 30 - 35 days after the last dose of VEDVsPayload. All patients that discontinue Investigational product and have not withdrawn consent to participate in the study, will continue in the long term follow-up phase every 3 months.

Leaky gut is a possible precipitant of inflammatory bowel diseases. We determine whether leaky gut can be identified in patients with Crohn's disease and ulcerative colitis in comparison with non-IBD controls using confocal endomicroscopy, which can identify microscopic barrier function of the bowel mucosa. Patients are followed longitudinally to determine if they improve or flare requiring treatment escalation.

The study aims to develop and improve the Lidcombe Program, a treatment designed specifically for stuttering in preschool children. The aim of this project is to find out if all components of the Lidcombe Program are necessary to achieve stutter-free speech. We hope the information gained from this project will help speech pathologists provide improved treatment outcomes for children who stutter.

Osteoarthritis affecting the first metatarsophalangeal joint of the foot is a common condition which results in pain, stiffness and impaired ambulation. Footwear modifications and foot orthoses are widely used in clinical practice to treat this condition, but their effectiveness has not been rigorously evaluated. The aim of this study is to compare the effectiveness of rocker-sole footwear and individualised prefabricated foot orthoses in reducing pain associated with first metatarsophalangeal joint osteoarthritis.

Streptococcus pneumoniae is the commonest cause of pneumonia in the community, and a major cause of illness and death in the elderly. A free pneumococcal polysaccharide vaccination (PPV) program was introduced, in Australia, in 2005. However, responses to PPV in the elderly are often poor. A more immunogenic pneumococcal conjugate vaccine (PCV-7) is available for children under 2 but is not licensed for adults. We completed a study that compared the immunogenicity of PCV-7 and PPV in the hospitalised elderly who are at highest risk for pneumococcal disease. Elderly patients admitted to hospital that had not previously received PPV, were recruited and randomised to receive PCV-7 or PPV; those who received PCV-7 received PPV six months later. Results showed no significant differences in response after one dose of each vaccine; severely frail patients have less vigorous response to either vaccine. Final follow up was done at 12 months. To our knowledge there are no data on long term immunogenicity studies beyond 12 months comparing PCV and PPV responses in the elderly. The proposed study is a longer term follow up study of subjects five and ten years after the 12 month follow up period of a completed trial of pneumococcal vaccines in frail elderly people, to determine persistence or waning of immunity in surviving subjects as well as measuring influenza vaccine antibody. It is impractical to measure efficacy in such a group in a clinical trial, due to the low incidence of pneumococcal disease, so proxy measures of efficacy using immunologic surrogates are necessary. The surviving study subjects from the above mentioned study will be contacted and interviewed, a blood sample will be collected from them at their current residence to determine persistence or waning of immunity in surviving subjects as well as measuring influenza vaccine antibody. We seek permission to access hospital medical records to be able to determine rates of readmission and other clinical outcomes of older people and ascertain predictors of readmission. Access to the death register will enable us to determine death date and cause and ensure that no attempts are made to contact deceased participants. Following ethics approval, we will check hospital records to identify any further deaths in the trial cohort. For those patients still thought to be alive, we will then contact their last known GP, and check on vital status and medical presentations not resulting in hospitalisation and vaccinations received. If the GP believes the patient is still alive, we will write to them giving them notice that we wish to contact them to ask them to consider being included in a new long-term follow-up cohort study. In this initial letter we will enclose the patient and Responsible Person information leaflets with the consent form and a pre-paid envelope for them to consent or decline further involvement. There is limited available long-term follow-up data on antibody levels in older persons who have been vaccinated with conjugate pneumococcal vaccines.

This trial will compare three intravenous anti-inflammatory regimens against placebo in women undergoing hysteroscopy and dilatation and curettage (D&C) as a day case surgery. It will compare the effectiveness of pain relief immediately post surgery and during the subsequent 24 hours. It will also compare the incidence and severity of side effects of pain relieving drugs including nausea and itch, and overall quality of recovery from surgery using a well validated questionnaire.

Brief summary: Obstructive sleep apnoea (OSA) is the most common form of pathological breathing in sleep. It is characterised by frequent upper airway collapse, recurrent brief arousals that severely disrupt sleep, and marked repetitive oxidative and cardiovascular system stress. OSA has serious adverse health and quality of life consequences, including severe daytime sleepiness, cognitive impairment, a 2-fold increased risk of motor vehicle and other accidents, and hypertension. Of those patients diagnosed with OSA around 30% exhibit clinically significant symptoms only when supine. Although a number of supine avoidance therapies are available and generally effective (e.g. classic tennis ball devices designed to make supine sleep uncomfortable), all have problems that limit clinical utility. In a follow-up survey of patients established with a traditional tennis ball device, we found that over 90% of patients stopped using treatment within a median time of 1 month, primarily due to excessive discomfort. The acceptability of supine-avoidance therapy as a mainstream treatment for appropriately selected patients relies upon substantiating practical, acceptable and low cost methods to discourage supine sleep. The BuzzPOD (Gorman ProMed Pty. Ltd) is a position monitoring device, worn on the chest, employing a vibration function designed to discourage supine sleep whilst minimally disturbing the patient and bed partner. A previous small, randomised, controlled, cross-over pilot study of active versus inactive treatment (1 week of each with 1 week washout) in 14 patients confirmed that the device almost completely abolished the supine posture and usefully improved OSA severity. Adherence to treatment was high. Total use over the full 3 week trial was (mean+/-SD) 85%+/-23% of nights, 6.8+/-2.3 h per night including zero hours when not worn, and 8.0+/-1.2 h on nights when worn. These average nightly compliance values are around twice those typically reported for CPAP treatment in mild OSA (3.5+/-2.1 h/night). Compliance was maintained at that level for several months in several of the patients who agreed to a longer period of post-study surveillance. Thus, we believe there is now sufficient evidence to support a more rigorous longer-term trial of supine-avoidance therapy. The study intends to recruit 140 patients with supine-predominant obstructive sleep apnoea. Patients will receive both treatments in a randomly allocated order; the control arm will be standard, clinical, best-practice CPAP treatment, the experimental arm will be receive BuzzPOD supine-avoidance therapy. Patients will receive each treatment for 8 weeks, and will attend follow-up assessments just prior to concluding each treatment. It is expected that supine avoidance therapy in patients with positional OSA will achieve improvements in daytime sleepiness and quality of life that are not inferior to those following CPAP, and be better tolerated than conventional CPAP treatment.

This study aims to investigate the effectiveness of an interdisciplinary student clinic program for people with chronic disease compared to people who receive an oral health student-led clinic intervention. It is expected that people who receive the interdisciplinary clinic program will have better health and functioning, less disability, fewer hospitalisations, shorter lengths of stay, less ambulance usage and lower health costs than people who attend the oral health program; when people in the groups are matched using similar characteristics such as age, gender and number of comorbidities.

The primary purpose of this study is to compare the relative effectiveness of two different and standardised exercise programs (The Lyn Watson Program and The Rockwood Program) on function and instability specific outcomes as well as strength and scapula measures, on patients with non traumatic Multidirectional Instability of the glenohumeral joint. The hypothesis is that the Lyn Watson program will produce clinically and statistically superior results on outcomes due to the program’s focus on achieving and maintaining scapula control.