ANZCTR search results

The purpose of this research is to assess the safety, tolerability and effectiveness of bitopertin in patients with EPP and XLP. This will be the first time it has been given to individuals with EPP/XLP. In this study, up to 22 patients will be enrolled in Australia only. Erythropoietic protoporphyria and XLP are rare diseases which cause severe light sensitivity. Both are caused by mutations in the genes that produce enzymes of the heme production pathway. These defects lead to abnormally high levels of protoporphyrin IX (PPIX) in red blood cells, which leads to painful reactions when exposed to light. Additionally, PPIX accumulates in the liver and gall bladder and can cause gallstones and liver impairment. Current therapies for EPP/XLP are aimed at managing symptoms. At present, none act to reduce the high levels of PPIX in the blood. Increased PPIX is thought to be the underlying cause of the severe and potentially life-threatening effects of EPP/XLP. Thus, there remains a significant unmet medical need for new therapies which address the underlying causes of the disease. Disc Medicine Inc is developing bitopertin, an inhibitor of glycine transporter-1 (GlyT1) as a new treatment for EPP/XLP.

In this randomised, double-blind, placebo-controlled study, 80 adults experiencing anxiety will be randomly assigned to receive tablets containing either an echinacea extract (EP107) (40mg twice daily) or a placebo for 2 weeks. We will assess changes in anxiety using the State-Trait Anxiety Inventory and general wellbeing using the World Health Organisation (WHO) - 5 Wellbeing Index which will be administered at regular times before, during, and 1 week after tablet intake.

This study will determine safety and feasibility of therapeutic FMT for steroidrefractory (SR) and steroid dependent (SD) acute gastrointestinal (GI) Graftversus-Host Disease (GVHD) ocurring in patients after allogeneic bone marrow transplantation at Royal Brisbane and Women’s Hospital. It is a phase I/II open label, pilot study and will treat 10 participants. FMT is an effective therapy to restore the healthy GI microbial flora in recurrent Clostridiodes difficile (rCDI) gastroenteritis. There is emerging evidence of efficacy for FMT in GI-GVHD, an immune-mediated complication of bone marrow transplantation. FMT offers a novel and cost effective therapeutic option to reduce GVHD mortality and the morbidity associated with other secondary therapies, i.e. broad spectrum immunosuppressive treatments including Antithymocyte globulin (ATGAM). FMT will be offered in combination with standard of care including Ruxolitinib and following first line treatment with steroids. Efficacy of FMT as a therapy for SR and SD GI-GVHD will be assessed as a secondary endpoint. Laboratory samples for correlative scientific analysis will be collected to explore the mechanistic basis for therapeutic efficacy of FMT. The FMT product will be supplied by Australian Red Cross Lifeblood Microbiome, who are a TGA licenced manufacturer of voluntary donated human stool derived faecal Microbiota for transplant.

The aim of the conceal trial is to determine the mechanism underlying phantom limb pain in lower limb amputees using targeted muscle re-innervation and functional brain MRI. Patients will be randomised to TMR or medical management groups in a 2:1 ratio and undergo functional brain MRI pre-operatively and 12 months post-operatively (12 months apart if they are randomised to the medical management group). They will also complete pain and function questionnaires NRS, PROMIS, Neuro-QOL pre-operatively and 1, 3, 6, 9 and 12 months post-operatively.

Transitioning into residential aged care (RAC) can be a stressful period for some older adults. This study will investigate the effects of a physical activity (PA) program introduced to new residents within their first two weeks in RAC. In line with recommendations from the Royal Commission into Aged Care Quality and Safety to promote wellness and reablement among older adults, the aim of the study is to investigate the effect of an individually tailored PA intervention on the psychosocial and physical well-being of older adults who have recently transitioned into RAC. The exercise physiologist intervention will take place over 12 weeks and consist of both one-on-one and group exercise sessions. Participants will be recruited from a number of RAC facilities in Perth, Western Australia. The two-arm intervention will address the question: What is the effect of a prescribed exercise program on the psychosocial well-being of older adults transitioning into RAC, as measured by the Warwick-Edinburgh Mental Well-being Scale, the General Belongingness scale, and the Three-item UCLA Loneliness scale? It is anticipated that research findings will contribute to a more coordinated approach to wellness and reablement among older Australians and provide aged care providers with the evidence they need to replicate similar programs, especially during a time that is traditionally perceived as challenging for the resident.

Introduction The pelvic floor has a critical role in resisting intra-abdominal pressure and ground reaction force to maintain pelvic organ support and continence. Pelvic floor disorders (PFD), such as pelvic organ prolapse, urinary incontinence, or pelvic floor pain may arise when this system is compromised. Performing artists are a unique population group that may be exposed to factors potentially provocative of PFD and symptoms. Pelvic floor disorders and symptoms are posited to negatively impact exercise and performance participation. The purpose of this study is to describe the prevalence, impact and factors associated with PFD in performing artists. Methods and analysis This study is a cross-sectional survey, with a proposed sample size of at least 384 performing artists. Questionnaires will be distributed to performing arts organisations (recreational, professional and schools) across Australia via REDCap and all data will be anonymised. Data will be collected on the primary outcome, prevalence of PFD, using existing validated questionnaires. Data will also be collected on the impact PFD have on performance, training, and everyday activities and what factors that may be associated with PDF and symptoms (secondary outcomes). Data will be analysed and reported as frequency and percentage for prevalence. Associated factors will be explored using binary logistic regression, and descriptive analysis will be used to report on the impact of PFD in performing artists. Ethics and dissemination The study is deemed low-risk and La Trobe University ethics approval will be sought prior to commencement. Results will be disseminated via peer-reviewed publications, presentations at national and international conferences, and summary findings will be provided to Australian performing arts organisations and companies.

A pressure injury, or pressure ulcer, is localised damage to the skin and/or underlying tissue caused by unrelieved pressure, shear or friction. Some hospital patients develop pressure injuries- with those in intensive care units at increased risk because they are critically ill and immobile. Intensive care unit (ICU) patients are among the most vulnerable patients in hospital, placing them at very high risk of developing sacral (tailbone) pressure injuries (PI) or bed sores. PI prolongs hospitalisation for patients and increases healthcare costs. Patients report PI are painful and compromise their quality of life. Several specialised dressings prevent sacral PI however limited effectiveness evidence results in clinicians asking, ‘which dressing is better’? This ICU pilot study will determine the feasibility of launching a larger randomised controlled trial testing two prophylactic sacral dressing and provide clinicians with a definitive answer to inform their practice. The study hypothesis is to evaluate the feasibility of achieving participant recruitment (50% eligible and 70% recruited, with 90% retention), protocol fidelity (95% participants will receive the correct intervention) and less than 10% missing data.

The purpose of this study is to treat sleep difficulties in adolescents and young adults with cancer. Who is it for? You may be eligible for this study if you are aged between 16 to 25 years, and have been diagnosed with cancer. Study details All participants will be asked to complete questionnaires on their sleep. Based on the results of the questionnaires, each participant may either: not receive any treatment on sleep difficulties; receive educational materials which includes a 63 page self management resource on key cognitive behavioural techniques to manage sleep difficulties. This includes: practical advice on how to establish good sleep hygiene habits and use relaxation techniques to promote sleep; strategies to manage common worries and thoughts that interfere with sleep; as well as strategies to manage common cancer treatment side effects that interfere with sleep. Participants are given a coaching call 3 weeks after receiving the self management resource and their sleep difficulties are reassessed 5-weeks after receiving the the self management resource. If their sleep difficulties have not resolved post receiving the self management resource, the participant will be invited to attend 4 x 50-60 minute weekly therapy sessions to address their sleep difficulties. It is hoped to this research will determine if this stepped care program is a feasible way to treat sleep difficulties in adolescents and young adults with cancer.

This is an observational study which aims to see if levels of circulating tumour cells (CTCs) mobilised during curative-intent radiotherapy (RT) for non-small cell lung cancer (NSCLC) can be used to predict disease progression and development of metastases. Who is it for? You may be eligible for this study if you are aged 18 years or older and you have been deemed eligible to receive RT for NSCLC after positron emission tomography (PET) staging. Study details All participants will have collection of blood samples to measure the level of CTCs, pre-treatment, up to 3 pre-defined intervals (24 hours after commencement of radiotherapy, midway through radiotherapy, and final week of radiotherapy) throughout treatment and at disease assessment. A further sample will be taken at the time of suspected relapse, should this occur. These samples are collected on days participants are already attending the hospital. This will then be analysed to determine the correlation with survival, metastasis, local disease progression, and thromboembolic events (i.e. the development of blood clots) as determined by a review of medical records. The DNA of CTC samples will also be assessed to determine whether this influences any of the above parameters. Participants will be followed up in clinic as per standard of care requirements at 3 monthly intervals from completion of radiotherapy (or more often if required clinically) until last patient enrolled completes one year follow up. It is hoped that this study may show whether changes in CTC or CTC DNA levels after radiotherapy may have prognostic significance. Although this will not influence your current treatment, this may be used to inform the treatment of patients with NSCLC in future.

Cancer Services at South Western Sydney Local Health District (SWSLHD) have a diverse spectrum of patients with 18.5% of patients coming from a culturally and linguistically diverse (CALD) background. However, previous investigations in this cohort determined that trial participation was significantly lower in CALD patients (5.7%) compared to non-CALD patients (8.4%). The radiation oncology department at SWSLHD has a number of recruiting clinical trials that involve investigations surrounding the incorporation of magnetic resonance imaging (MRI) into routine radiotherapy planning and delivery. Literature indicates that barriers to CALD participation in clinical trials include communication and health literacy concerns. Therefore, the aim of this study is to assess the feasibility of providing translated educational materials to CALD patients, and the effect that this has on CALD patient recruitment to clinical trials of MRI in radiotherapy. Who is it for? You may be eligible for this study if you are aged 18 years or older, are attending the Liverpool, Campbelltown, or Bankstown Cancer Therapy Centre Clinics for a Radiation Oncology Consultation, and are of Chinese, Arabic or Vietnamese background requiring interpretive services. Study details All participants will receive educational materials that are translated into their native language. The materials will be delivered face to face, and will include a 5-minute video and a brochure containing standard patient MRI information. Before and after receiving these materials, participants will be administered a survey to assess their willingness to participate in clinical trials of MRI in radiotherapy. The recruitment rate of CALD patients to these trials will also be assessed at the end of the study, as well as the influence of the educational materials on recruitment rates. It is hoped that this study may show that providing translated educational materials to CALD individuals is feasible, and may improve rates of recruitment to clinical trials in this population.