ANZCTR search results

This study aims to determine whether there is measurement equivalence between traditional paper-and-pen and electronic delivery of disease-specific patient-reported outcome measures (PROMs) in paediatric burn scar patients and their caregivers. Determining the equivalence of these delivery methods will provide evidence regarding the ability to transition from paper-based administration to electronic based administration of patient-reported outcome measures involving children and caregivers in research and clinical practice based on the comparability of psychometric properties and conceptual base of the measures. We hypothesise that there will be acceptable equivalence between pen-and-paper and electronic completion methods.

The objective of this study is to provide supporting clinical evidence in particular with regard to the efficacy and safety of the ElePulse electroporation ablation system. It might reveal indicators for hidden risks and trigger further dedicated investigations or tests.

The availability and accessibility of post-diagnostic support for people living with dementia and their carers are of central importance in dementia care. People with dementia and their carers benefit from timely access to emotional and practical support, lifestyle advice, and meaningful activities to maximise their quality of life and potentially delay cognitive decline. Allied health professionals at Canberra Health Services at the University of Canberra Hospital and the University of Canberra have designed an evidence-based 12-week multi-component program, tailored to people with dementia and their carers, which includes: physical activity, social engagement, nutrition assessment, education and capacity building. The team has engaged with Dementia Australia advocates to refine the program and set priorities for research. The research study will assess the value of a pilot version of this multi-component dementia support program, measure impact and effectiveness for people with dementia and their carers. If shown to be successful, the multi-component program could become part of standard care in the ACT region and provide a replicable model of comprehensive post-diagnostic services for dementia care Australia-wide.

Patients with Ischaemia and No Obstructive Coronary Artery Disease (INOCA) are under-diagnosed and under-treated in current clinical practice. There is a growing body of evidence that pathological abnormalities in coronary vessels characterise these conditions, leading to increased morbidity and mortality. These patients continue to experience recurrent angina, a poor quality of life and adverse cardiovascular outcomes. Although there is growing recognition of the impact of this entity amongst the cardiology community, methods and criteria pertaining to the diagnosis remain poorly defined. The MAD-NOCA trial (Multivessel compared to Single-vessel Functional Angiography to Diagnose Patients with No Obstructive Coronary Artery Disease) aims to develop a novel diagnostic algorithm for patients with INOCA. This randomised controlled trial will evaluate whether comprehensive three-vessel coronary physiology and vasoreactivity testing, compared to standard single vessel testing, in patients with INOCA, will: 1) Increase the proportion of patients diagnosed with a ‘disorder of coronary vasomotion’, 2) Influence clinical management, 3) Lead to improved clinical outcomes

This is a Phase 1b, parallel single-dose study to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of tinlarebant when administered as an oral dose to healthy volunteers aged 50-85. Tinlarebant (previously called LBS-008) is being developed as an oral treatment to slow or halt the progression of dry age-related macular degeneration (AMD) and juvenile onset macular degeneration, known as Stargardt disease (STGD). Accumulation of lipofuscin bisretinoids in the retina is associated with several retinal degenerative diseases including dry AMD and STGD. A total of up to 16 healthy volunteers aged 50-85 are planned to be enrolled into 2 cohorts (8 participants per cohort). Participants will receive a single oral dose of tinlarebant, either 5 mg (Cohort 1) or 10 mg (Cohort 2). The total maximum study duration for participants in this study is 45 days, inclusive of visit windows. This includes a screening period of up to 27 days (Day -28 to Day -2), confinement to the clinical facility over 2 nights (Day -1 to Day 2) and 5 outpatient visits including the EOS visit or Day 15. Tinlarebant will be administered in tablet form; a single tinlarebant tablet will contain 5 mg of IP. Participants in Cohort 1 will receive 1 tinlarebant tablet (5 mg dose) and participants in Cohort 2 will receive 2 tinlarebant tablets (10 mg dose). Enrolment and dosing of participants in Cohorts 1 and 2 may occur concurrently. Healthy volunteers aged 50-85, will be screened between Day -28 and Day -2, inclusive. Participants will be admitted to the clinical facility on Day -1 with tinlarebant dosing to occur on the following day (Day 1). All participants will be dosed under fasted conditions. Participants will be confined at the clinical facility from Day -1 through to Day 2 with discharge following completion of all PK and PD blood sample collections and other scheduled assessments on Day 2. If participants experience any clinically significant (CS) AEs during the confinement period, they may remain in the clinical facility for further observation at the discretion of the Principal Investigator (PI). Blood samples for PK and PD analysis will be collected pre-dose and at timepoints up to 24 hours (Day 2) post-dose during the confinement period. Participants will be required to attend the clinic as outpatients on Days 3, 4, 6 and 8 for further safety assessments, PK and PD blood sampling. Participants will then return to the clinical facility for final safety assessments, In the event of early study termination prior to the EOS visit (Day 15), participants will be requested to attend the clinic for an early termination (ET) visit.

This is a two-part, double-blind, placebo-controlled, Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple ascending doses of MVD1 in healthy volunteers. The decision to escalate between dose levels and proceed from Part A to Part B will be based on review of the safety and available PK data by the study safety review committee (SRC) from a minimum of 5 active treated participants per cohort. The SRC may recommend repeat of a cohort, or a change in the dose levels of subsequent cohorts, or that no further dose escalation cohorts occur and recommend the final sample size of the MAD cohorts based upon consensus and emerging data from safety, PK and PD biomarkers. In Part A, screening for study eligibility will occur between Day -28 and Day -2 inclusive. Following confirmation of eligibility on Day -1, participants will be randomised to be enrolled in Cohorts S1, S2 or S3 and to receive either MVD1 or placebo. Participants will then be admitted to the clinical facility. The confinement period will commence on Day -1, followed by dosing on Day 1 with placebo or active drug in a double-blind manner. Participants will receive a single oral dose of MVD1 or placebo on Day 1. Participants will be discharged from the clinical facility on Day 4 following completion of study assessments and will be required to attend the clinic for a final follow-up visit on Day 8 ± 1 Day. In Part B, screening for study eligibility will occur between Day -35 and Day -2 inclusive. Following confirmation of eligibility on Day -1, participants will be randomised to be enrolled in Cohorts M1, M2, or M3 and to receive either MVD1 or placebo. Participants will then be admitted to the clinical facility. In Part B, the confinement period will commence on Day -1, followed by dosing on Day 1 with placebo or active drug in a double-blind manner. Participants will receive twice daily oral doses of MVD1 or placebo (administered 12 hours apart) on Days 1-14 and a single dose on the morning of Day 15 (total of 29 doses). Participants will be discharged from the clinical facility on Day 18 following completion of study assessments and will be required to attend the clinic for a final follow-up visit on Day 22 ± 1 Day.

This experimental study aims to investigate the effects of presenting diagnostic accuracy information from real-world studies, and of using health literacy sensitive text for RAT instructions, on people’s decisions to self-isolate and take other preventative measures. Australian adults aged 18 years and over will be sampled. They will be presented with 5 different hypothetical scenarios and asked a number of survey questions investigating knowledge, understanding, decision making, risk perception and behaviour to prevent onward spread. RAT instructions that are easier to understand and include real-world estimates of accuracy may help users to interpret RAT results and to take appropriate actions.

When conservative treatment fails for constipation or faecal incontinence, biofeedback and pelvic floor health therapy are two different management options. These two modalities are often thought to be similar by some physicians due to both employing components of pelvic exercise. However, these two modalities have very distinct programmes and are usually delivered by different health care professionals (nurses versus physiotherapists). Despite this, no randomised controlled trial has been performed to date to compare them for both indications. This study aims to investigate any difference in outcome between the two modalities.

Older drivers have relatively high crash rates and are increasing in number. We aim to evaluate the relative effectiveness and cost-efficiency of interventions for older drivers over a 12 month period. A randomised control trial comparing driving lessons, personalised feedback on driving skill, and a road-rules refresher workshop will be conducted. If effective, interventions will improve driving safety, reduce costs associated with crashes, and maintain social participation.

The aim of this study is to assess the feasibility, safety, and efficacy of autologous GD2-specific chimeric antigen receptor-expressing peripheral T cells (GD2-CAR T cells, a blood transfusion derived from the patient’s own cells) in patients with recurrent GD2-positive glioblastoma multiforme. Who is it for? You may be eligible for this study if you are aged 18 years or older, you have histologically confirmed glioblastoma that is recurrent, and your tumour stains positively for the marker GD2 on biopsy. Study details All participants will undergo collection of peripheral T cells by apheresis (i.e. removing whole blood from a vein) to manufacture the GD-2 CAR T cells. If manufacture of the therapy is successful, the participant will receive a single treatment of GD2-iCAR-PBT intravenously at an initial dose of 1 x 10^8 cells/m^2. For 8 weeks following the GD2-CAR T cell infusion, participants will be assessed for any toxicities from the treatment, and at 8 weeks post-injection their initial tumour response will be assessed using brain MRI. If determined to be safe and effective, subsequent participants enrolled into the study may receive a higher starting dose of GD2-iCAR-PBT, to determine the maximum safe dose of administration. All participants will be monitored for up to 1 year post-enrolment for efficacy of the treatment using brain MRI. It is hoped that this study may help us find the dose of administration of GD2-CAR T cell therapy that produces the greatest tumour response with the least toxicities for the treatment of glioblastoma multiforme. This may help to direct treatment of other patients with this tumour in future.