ANZCTR search results

Medulloblastoma is a rare brain malignancy, mainly affecting children. Treatment of this rapidly growing tumor begins with maximal surgical removal plus radiation and chemotherapy. Treatment toxicity is high. Post-pubertal and pediatric medulloblastomas are biologically and prognostically different, which mandates age-adapted treatment strategies. Patients after puberty bear an intermediate to high prognostic risk. This means that a large number of these patients, are faced with death and/or disability (mainly neurocognitive). Therefore, the scientific and medical need is high. One of the genetic subgroups of medulloblastoma, the SHH-subgroup (Sonic HedgeHog- subgroup), is highly overrepresented in medulloblastoma patients after puberty. This subgroup can be treated with a targeted therapy. The investigators will therefore randomize patients and treat SHH-subgroup patients with sonidegib and a reduction of radiotherapy dose in the experimental arm of the trial. The hypothesis that this personalized risk-adapted therapy will improve outcomes in view of increased efficacy and decreased toxicity.

Myelodysplastic Syndrome (MDS) is a group of disorders that gradually affect the ability of a person's bone marrow (semi-liquid tissue present in many bones like backbones) to produce normal blood cells. Some people with MDS have a risk of the disease progressing to acute myeloid leukemia (AML), and a risk of death from the disease itself. Symptoms of MDS include fatigue, shortness of breath, unusual paleness due to anemia (low red blood cell count), easy or unusual bruising, and red spots just beneath the skin caused by bleeding. The purpose of this study is to see how safe and effective venetoclax and azacitidine (AZA) combination are when compared to AZA and a placebo (contains no medicine), in participants with newly diagnosed higher-risk MDS. Venetoclax is an investigational drug being developed for the treatment of MDS. The study consists of two treatment arms - In one arm, participants will receive venetoclax and AZA. In another arm, participants will receive AZA and placebo. Adult participants with newly diagnosed higher-risk MDS will be enrolled. Around 500 participants will be enrolled in approximately 220 sites worldwide. Participants in one arm will receive oral doses of venetoclax tablet and intravenous (infusion in the vein) or subcutaneous (given under the skin) AZA solution. Participants in another arm will receive oral doses of placebo tablet and intravenous or subcutaneous AZA solution. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the course of the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood and bone marrow tests, checking for side effects, and completing questionnaires.

Patients who are critically ill with COVID-19 requiring life support in an intensive care unit (ICU) have increased risk of morbidity and mortality. Currently the ICU community does not know what effect the disease, the ICU admission, physiotherapy interventions and life support have on their long-term quality of life and whether they can return to their pre-illness level of function following ICU. COVID-Recovery will describe the physiotherapy interventions delivered to critically ill patients with COVID-19. In survivors, COVID-Recovery will utilise telephone follow-up of ICU survivors to assess disability-free survival and quality of life at 6 months after ICU admission. Additionally, COVID-Recovery will identify if there are predictors of disability-free survival. COVID-Recovery will aim to select up to 300 patients diagnosed with COVID-19 from ICUs in Australia. If they survive to hospital discharge, patients will be invited to receive a telephone questionnaire at 6 months after the ICU admission that aims to assess their long-term outcomes, including physical, cognitive and emotional function, quality of life, and whether they have been able to return to work following ICU discharge. To describe the experience of critical illness in survivors of COVID-19 and their family members. To explore and describe functional recovery, respiratory system function and respiratory health morbidity up to 6 months after ICU admission in persistently critically ill adults with COVID-19

This is a multi-site, prospective, non-randomised trial assessing the implementation of a smartphone application-based model of care for patients with COVID-19 infection managed in community isolation. We will recruit 2000 COVID +ve patients aged 18 years and over who are managed at home. The objective will be to describe the rates of avoidable presentations to ED and 30 day all case mortality per diagnosed COVID-19 case and to compare these to a propensity matched and synthetic control group.

This study aims to implement a targeted 4CMenB immunisation program in young people aged 14-19 years in the Northern Territory (NT). As part of the NT program consenting 14-19 year olds will receive 2 doses of the licensed 4CMenB vaccine. An oropharyngeal swab will be collected on the same day as the first dose of the vaccine and 12 months later to assess carriage of Neisseria meningitidis. The first swab will assess baseline carriage prevalence among 14-19 year olds in the NT. The swab taken 12 months later will provide data on the change in carriage that may occur after implementation of the immunisation program. Emerging evidence suggests that the 4CMenB vaccine may be protective against gonorrhea. Therefore, vaccine effect (impact and effectiveness) against both invasive meningococcal disease (IMD) and gonorrhea in the NT will be assessed using data from the above study comparing notifications between vaccinated and unvaccinated as well as comparing pre and post implementation periods.

There is ample evidence showing that loneliness is a public health problem that influences social, mental and physical health. The current project is about evaluating the effectiveness of the Kindness Is NextDoor (KIND) Challenge, a social networking platform aimed at reducing loneliness and social isolation in specific Nextdoor communities within Australia. As the landscape through which people make social connections changes due to technology it is important to evaluate the effectiveness of platforms that are designed to reduce loneliness within communities. This is the first study of its kind in Australia and will help provide insight into how to target social isolation and loneliness within communities using social networking platforms.

This Phase 2a study will assess the safety, antiviral activity, and pharmacokinetics (PK) of ABI-H2158 administered once daily for up to 72 weeks in combination with entecavir (ETV) in participants with chronic hepatitis B virus (HBV) infection.

A Phase 2a, multicenter, 4-part, randomized, double-blind, dose-ranging, placebo-controlled study to evaluate the safety, tolerability, and PK of once-daily treatment with PLN-74809 in participants with idiopathic pulmonary fibrosis.

COVID-19 is a global pandemic. So far encouraging results have been shown in different parts of the world with the utilisation of hydroxycloroquine, zinc, and azithromycin, and early studies into some of these, plus some with Vitamin C, have also proven beneficial. Vitamin D levels have also been shown to be an important indicator to the severity of symptoms in COVID-19 patients.

The primary objective of this study is to determine if left atrial appendage closure (LAAC) with the WATCHMAN FLX device is a reasonable alternative to non-vitamin K oral anticoagulants in patients with non-valvular atrial fibrillation.