ANZCTR search results

Phenylketonuria (PKU) is a rare Inborn Error of Metabolism (IEM) with an incidence of approximately 1 in 10 - 15,000. Prior to the advent of newborn screening and effective dietary treatments, PKU caused a severe neurodevelopmental phenotype marked by global developmental delay, severe intellectual impairment, and seizures. Early diagnosis from newborn screening and introductions of dietary interventions prevent these severe neurological manifestations. Despite this, neuropsychological complications of PKU are observed when control is suboptimal, which include reduced executive function, attention deficit, decreased processing speed, cognitive impairment and disturbances in emotion and behaviour. Neuro-disabilities are thus a key phenotype amongst the PKU cohort when their metabolic control is suboptimal. This project is aimed at exploring practical tools to improve our management outcomes for children and families living with this rare chronic disease. We hypothesise that participating in a brief evidence-based parenting intervention (Triple P), when compared to baseline, will improve progress towards child behaviour/parenting goals, improve children’s quality of life, decrease parent-reported use of ineffective parenting practices, increase parent self-efficacy, reduce parent-reported child behavioural problems, reduce parenting stress, and improve children’s blood Phe levels.

This pilot randomized controlled trial (RCT) aims to examine the feasibility, efficacy, and cost-effectiveness of a new risk-targeted transdiagnostic CBT telehealth program (FullFix) for comorbid alcohol and other drugs (AOD) and mental health problems in young people. The study aims to recruit 130 participants aged 16 - 25, who will be randomised to receive 4 core modules (and up to 4 additional modules) targeting transdiagnostic risk and protective factors for mental health and AOD use, or usual AOD care. Participants' AOD use, mental health, and functional outcomes will be assessed at baseline, and at 1 month, 2, 6, and 12 months post baseline.

This study aims to investigate whether a 12-week combined psychoeducation and home-based exercise intervention can improve dementia risk, mood, health knowledge, cognition, sleep, metabolic markers and brain connectivity in older adults with cognitive difficulties. The active intervention will comprise an individually prescribed, home-based exercise and psychoeducation program. Results will be compared to an active waitlist control group, who will be offered the psychoeducation program at the end of the trial.

The purpose of this study is to examine the action of a new medication (called “(Z)-endoxifen”) in patients with breast cancer. Who is it for? You may be eligible for this study if you are aged 18 or older and are scheduled to undergo mastectomy or lumpectomy for invasive breast cancer Study details All participants in this study will take oral capsules of the study medication for at least 14 days and up to 30 days prior to day of their surgery. Before and after treatment, participants will provide a tumour biopsy and blood sample. Additionally, participants will need to consent to their removed tissue being tested. It is hoped this research will provide information regarding the action of (Z)-endoxifen and potentially lead to new therapeutics for breast cancer.

The aim of this study is to investigate the effects of single dose Prickly Pear (Opuntia ficus indica) fruit juice consumption, standardised for Total betalain Content (TBC), on CVD risk factors in postprandial blood lipids and physiological responses in healthy males. This double-blind placebo-controlled trial will model the potential blood lipid lowering effect of Prickly Pear fruit juice in combination with a ‘meal-model’ (high-fat muffin) on CVD risk factors in healthy humans.

Creatine is a dietary metabolite responsible for buffering quick changes in cellular energy supply and demand. We hypothesise that maintaining a particular level of circulating creatine during pregnancy can help ensure proper development and function of the placenta, and assist in fetal growth and development. The Creatine and Pregnancy Outcomes Study - CPO - has been specifically designed to provide thorough understanding of maternal creatine metabolism across gestation, including the placenta's contribution to maternal and fetal creatine levels. We will also collect nutrition data to enhance our understanding of how differing dietary habits over pregnancy may impact on creatine levels, and if these differences affect pregnancy outcomes.

This study of RYI-018 will be conducted in 20 healthy participants with a BMI between 25.0 and 40.0 kg/m2. The participants will be randomized in to one of two treatment arms, each arm having 10 participants. The study will estimate the absolute bioavailability of a sub-cutaneous formulation versus an IV formulation, as well as assess the safety and tolerability of RYI-018. Participants will receive a single dose of RYI-018 (after an overnight fast of at least 10 hours) and will be confined for approximately 24 hours after RYI-018 administration. All participants will be followed-up through 90 days post-dose. It is hypothesised that the subcutaneous formulation will work in the same way as the intravenous formulation, allowing for easier administration of RYI-018 and subsequently higher treatment compliance.

The purpose of this unblinded, non-randomized, phase I/II trial in patients who require a cochlear implant, is to evaluate the safety and efficacy of localised cochlear neurotrophin gene therapy, by comparing patients receiving a cochlear implant (control group) and patients receiving the gene therapy in conjunction with the cochlear implant (treatment group). The study of the control and treatment groups will not be conducted in parallel because of the logistics around the audiometric assessment of the subjects, which requires extension of the study across a two-year time frame. All subject measures will be identical for both groups, as well as all trial procedures, apart from the treatment and surgical procedures surrounding the delivery of the neurotrophin-encoding DNA to the cochlea, which is an amendment to the normal cochlear implant surgery protocol. For the treatment group, at surgery, prior to insertion of the cochlear implant electrode array into the patient’s cochlea, a cochlear gene delivery electrode array is inserted, naked DNA is injected and then a brief train of electrical pulses lasting less than one second is used to deliver the DNA locally to the cells closest to the electrodes. DNA dispersed through the cochlear tissue, but outside this local electric field, is not taken up by cells and degrades. This somatic cell gene electrotransfer process adds about fifteen minutes to the surgery time. The cochlear gene delivery electrode array is withdrawn after the DNA delivery (BaDGE process) and the cochlear implant itself is then implanted as normal. Participants will be enrolled in the trial for a total of 52 weeks. Data will be collected in the form of completed case record forms, blood tests, questionnaires and adverse events, alongside audiological measurements. Subjects will be followed up at multiple centres for the duration of the trial. They will be recruited from NextSense, cochlear implant surgery will take place at the Royal Prince Alfred Hospital (Sydney), and follow-up visits will take place at both the Australian Hearing Hub (Macquarie University) and NextSense.

The purpose of this study is to assess whether an alternative method (called the ‘Alternative Pathway’) of offering the National Bowel Cancer Screening Program via primary health care centres can improve Indigenous participation rates. Who is it for? You may be eligible for this study if you are an Aboriginal and Torres Strait Islander person aged 50 to 74 who attends an Indigenous Primary Health Care Centre that is taking part in the study. Study details Potential screening participants will be randomised (by chance) into two groups depending on the health centre they attend. One group of centres (called Group A) will offer participants a bowel screening kit as part of the Alternative Pathway. The other group of centres (Group B) will also offer participants a bowel screening kit as part of the Alternative Pathway, however staff at these centres will have received additional training from the research team. Screening kits will be the same as those used in the usual method of offering the National Bowel Cancer Screening Program. Samples will be tested, results reported to participants and follow up of positive results will all occur within the national program as usual.. Reports from the National Bowel Cancer Screening Register and from Indigenous Health Centres will be analysed to see how many Indigenous people complete a bowel screening test offered through the Alternative Pathway, and whether extra training for health centre staff makes any difference to the screening rate. It is hoped this research will raise awareness and increase accessibility for Indigenous Australians to participate in the bowel screening program, enabling early detection of bowel cancer to improve survival outcome for Indigenous Australians.

Background: It is common for toddlers to display disruptive behaviors (e.g., tantrums, aggression, irritability) but when these become severe and persistent, they can be the start of a trajectory towards poor outcomes across the lifespan. Evidence indicates that treatments should be provided for these children as early as possible, i.e., in infancy and the toddler years, to provide the best opportunity for success. A number of attachment theory-based parenting intervention approaches targeting high-risk children and caregivers have been developed, but evidence of their ability to bring improvements in both attachment and behavioral domains in the specific age of toddlerhood (14-24 months) is limited. Parent Child Interaction Therapy (PCIT) is a popular and evidence-based intervention for children with disruptive behaviors. PCIT-T is a promising new adaption of PCIT, developed at the Karitane Toddler Clinic in South Western Sydney, designed to meet the specific developmental needs of toddlers aged 14-24 months. Integrating attachment and behavioral principles, PCIT-T aims to strengthen the quality of the parent-child attachment relationship and seeks to enhance parental capacity to assist emotional and behavioral regulation in the child. Study aims and method: This study will evaluate the efficacy of the PCIT-T intervention for toddlers aged 14-24 months with disruptive behaviors using a randomized controlled design. One hundred and fifty toddlers with parent reported disruptive behavior will be randomly allocated to receive either PCIT-T, Circle of Security Parent Training (COS-P; a popular attachment-theory based intervention designed to improve parenting and parent-child relationship) or to be in a waitlist control group. Primary outcome variables will include: child behaviour and compliance; child social-emotional functioning (including emotional regulation ability); and parent-child attachment relationship quality. Secondary outcomes will include parental emotional well-being and skill; parenting behaviour, attributions and child abuse potential; and child language. All participating parents will also be invited to take part in a semi-structured interview at the completion of the study to provide qualitative feedback about the program and their perceptions of child outcomes. Expected outcomes: Delivered in the early intervention period of toddlerhood, PCIT-T has the potential to bring about significant and lasting changes for some of society’s most vulnerable children. Results of this study will thus be of immense public health significance, and will be of interest to clinicians, researchers and policy makers both in Australia and internationally.