ANZCTR search results

Cardiac involvement remains one of the biggest killers in Fabry disease. Yet our ability predict cardiac involvement before irreversible damage occurs remains limited. The chief reason for this is the differences in Fabry disease presentation that remains unexplained by biomarker or predictable natural history. Current tools, such as biomarkers (e.g., enzyme activity level, plasma Gb3, etc.) and genetic testing give a good measure of 'general' Fabry progression or diagnosis; but these are non-specific to phenotype or individual organ involvement. Further insight into which patients are at risk of developing heart complications would direct clinicians to consider commencement of Fabry-specific therapy in potentially high-risk patients. Markers and mechanistic insights that explain individual susceptibility to cardiac fibrosis and hypertrophy in the context of Fabry disease may also have broader implications to understanding heart failure and cardiomyopathy more broadly.

This study is a prospective, randomized, longitudinal study of the clinical and biomechanical outcomes of osteoarthritis patients treated by two different alignment philosophies for total knee replacement. All patients will be treated with the same knee system, implanted using nagivated assisted technology. We hypothosize that Inverse Kinematic alignment (iKA) will result in better Oxford Knee score (OKS) and satisfaction visual analogue scale (VAS) score compared to mechanical alignment (MA).

This study builds upon our world-first clinical trial of nicotinamide in glaucoma (ACTRN12617000809336) demonstrating the potential protective role of vitamin B3 (nicotinamide) supplements in people with glaucoma. Glaucoma causes progressive loss of nerve tissue in the eye, and irreversible vision loss. This study investigates the effect of taking nicotinamide supplements over 2 years on the eye’s structure and function compared to placebo. The primary aim of this study is to determine whether nicotinamide supplements in participants with glaucoma leads to reduced rate of progression in visual function measured using visual fields over 2 years. Participants diagnosed and treated for glaucoma will be invited to undertake the study. They will be randomly assigned to take nicotinamide or placebo daily for 24 months. Clinical tests including visual fields and imaging of the eye are performed at baseline, and every 4 months post-intervention.

Patients with persistent or chronic pain often have underlying chronic conditions that can be overlooked such as anaemia, which can have significant effect on the quality of life of patients. A recent pilot study involving patients who have persistent pain for greater than 12 months have shown that 59% have iron deficiency. This study will follow on from the pilot study to examine if intravenous iron infusions do improve this cohort of patients fatigue levels, their health-related quality of life, pain levels and reduce pain medication use. This study will be conducted at a Quaternary hospital in Brisbane as a single-site, single-blinded, parallel-group randomised controlled trial. We plan to recruit 160 participants who will be randomised to receive either intravenous iron infusion or placebo. The outcomes will be measured using specific validated surveys and questionnaires related to fatigue (FACIT), health-related quality of life (SF-36) and pain (ePPOC).

This study is a randomised open-label trial of the Moderna Omicron-containing bivalent booster candidate mRNA-1273.214 (mRNA-1273.214) vaccine. Participants who have received 2-3 prior doses of the any COVID-19 vaccine will be randomised into two arms: to receive a 3rd dose of the Moderna Bivalent Omicron vaccine on enrolment or 3 months later. Both groups will be followed-up for six months after enrolment.. We hypothesise that there will be a difference in measurable SARS-CoV-2 antibody responses in people who receive a Moderna Bivalent Omicron COVID vaccine booster immediately on enrolment in the study compared to those who wait an additional three months before receiving a booster dose. A booster dose of the Moderna Bivalent Omicron r vaccine will be administered intramuscularly. One dose (0.5 mL) contains 50 micrograms of mRNA-1273.214 (25-µg each ancestral Wuhan-Hu-1 and omicron B.1.1.529 spike SARS-CoV-2 mRNAs) (embedded in lipid nanoparticles). This is a single-site study with interested potential participants attending the Royal Melbourne Hospital for recruitment, the consenting process and study visits. A total of 64 participants will be recruited (32 per arm).

Traditionally, combination therapy has not proven to be effective in Myelodysplasia (MDS) given the increased toxicity of drugs used in combination with standard therapies. The aim of this master platform trial is to test a range of novel treatments aimed at targeting MDS to find safe and effective drug combinations. You may be eligible to participate in this study if you are aged 16 or older (depending on the study, this may be limited to 18 and older) and you have a diagnosis of either MDS or acute myeloid leukemia (AML) with less than 30% blasts. If you choose to enrol in this trial you will undergo a screening process where blood and bone marrow samples are taken prior to starting any treatments. The study doctors will assess your samples for specific MDS markers and will then prescribe a combination treatment that may be effective. Participants who do not respond to treatment or whose disease worsens may be removed from treatment and be reassessed for the next best treatment option to receive. During treatment participants will be assessed regularly which will include physical exams, blood tests, ECG (heart monitoring), bone marrow biopsies, toxicities to the treatment, quality of life. After treatment, disease will continue to be monitored and if participants have MDS progression or morphological relapse they will be assessed for the next best treatment option. It is hoped that the results of this trial will help us to determine which drug combinations are safe (minimal side effects/toxicity) and effective for treating MDS.

This study aims to evaluate the effectiveness of a new online treatment for body dysmorphic disorder (BDD) in adolescents. BDD is an underrecognised and undertreated mental health condition, characterised by a preoccupation with perceived flaws in physical appearance. There is a paucity of research exploring the disorder among youth. In adults with BDD, cognitive-behavioural therapy (CBT) has been shown to be an effective treatment. There is some evidence that CBT can be effective for some youth with BDD, however many young people remain impaired by their BDD following treatment. Therefore, this study aims to test a developmentally-tailored CBT approach to treating adolescent BDD. The therapy will be delivered via videoconference to families, to increase accessibility to the study.

This is Part B of a 2-part, Phase 2, Clinical Trial Evaluating the Efficacy, Safety, Tolerability, and Pharmacokinetics of PRAX-944 in Adults with Essential Tremor. Each participant will complete 3 study periods: Screening, Treatment Period (21 or 28 days) and Safety Follow-up.

In recent years significant interest has been shown in the role of contact heat evoked potentials (CHEPs) in the assessment of spinal cord damage. A single study comparing CHEPs with dermatomal somatosensory evoked potential (dSSEP) and clinical sensory testing found CHEPs to be the most sensitive measure of spinal cord damage. In subjects with spinal cord injury (SCI), peripheral sensitisation with topical capsaicin was found to improve the detection of “silent” fibres using quantitative sensory testing (QST). Given that QST is a psychophysical test (dependent on patient report) more objective neurophysiological tests would be valuable. Several methods have been explored to improve the acquisition of potentials in patients with and without nerve damage. The two most promising methods are the use of chemicals to achieve peripheral afferent sensitisation and raising the baseline temperature of the stimulation. This study assesses whether increased baseline temperatures and peripheral sensitisation using topical capsaicin improves the detection of “silent” spinal cord fibre tracts using contact heat evoked potentials (CHEPs) in people with spinal cord injury (SCI) and below-level neuropathic pain (BLNP). Given the onset of SCI BLNP is often delayed identification of partially preserved fibres may be a risk factor that can be identified early in an individual. The detection of a potential peripheral contributor to the pain will also influence the treatment options pursued. Primary Objective: Determine whether CHEPs are able to detect subclinical spinothalamic fibre (STT) preservation following SCI. Secondary Objective: Determine whether CHEPs taken from an area of pain (below the SCI) are more frequently observed when peripheral sensitisation with capsaicin and baseline temperatures up to 42°C are used in subjects with clinically complete spinal cord injuries and BLNP.

The aim of this randomised controlled trial is to increase adoption of the lunchbox nutrition program ‘SWAP IT ’ in primary schools located in the Mid North Coast Local Health District. This trial will evaluate whether a theory-based multi-component dissemination strategy targeting barriers to adoption can improve the adoption rates of the nutrition program. Schools will be randomly allocated to receive either a multi-component dissemination strategy over a four month period, or a minimal intervention control group. Intervention effectiveness will be determined by comparing, relative to control, the absolute difference in the proportion of schools adopting the program at 6-month follow up. The trial aims to generate evidence regarding the effectiveness of strategies to inform broader dissemination of the lunchbox nutrition program.