Macquarie Park

Researchers are studying a treatment called MK-2214 to see if it can slow certain brain changes in people with early Alzheimer's disease (AD). AD is a form of dementia that causes memory loss, difficulties with communication, and challenges in decision-making, which affect daily activities. The study aims to find out if MK-2214 can slow the spread of tau protein in the brain compared to a placebo and to assess the safety and tolerability of MK-2214. Participants will receive either MK-2214 or a placebo through an intravenous (IV) infusion. The study is designed as a phase 2, randomized, placebo-controlled, double-blind trial with parallel groups. The treatment period lasts up to about 23 months, during which participants will receive infusions as scheduled. The placebo looks like the study treatment but contains no active drug, helping researchers understand the treatment's effects. Throughout the study, participants will be monitored for changes in tau protein levels in the brain using PET scans and for any adverse events or side effects. Researchers will track the number of participants experiencing adverse events and those who stop treatment because of them, with safety follow-up lasting up to approximately 26 months. Participants will also undergo brain imaging such as CT, PET, or MRI scans. The study involves regular assessments to measure the treatment's impact and ensure participant safety over the study duration.

Researchers are evaluating the safety and effectiveness of trontinemab in people aged 50 to 90 with early symptoms of Alzheimer's disease, ranging from mild cognitive impairment to mild dementia. This Phase III clinical trial focuses on those who show evidence of Alzheimer's pathology and have a recent history of cognitive decline. The study aims to measure changes in cognitive function over 72 weeks. Participants will be randomly assigned to receive either intravenous trontinemab or a placebo. The trial is designed as a double-blind, placebo-controlled study, meaning neither participants nor researchers know who receives the active drug or placebo. The treatment period lasts up to 72 weeks, during which participants will undergo various assessments to monitor their cognitive status and safety. During the study, participants will complete clinical tests including cognitive assessments and imaging such as MRI, PET scans, or cerebrospinal fluid analysis to confirm Alzheimer's pathology. A study partner will assist participants as needed. Researchers will track changes from the start of the study through week 72 using tools like the Clinical Dementia Rating. Safety monitoring and adherence to study procedures will also be closely observed throughout the trial.

Researchers are evaluating the potential neuroprotective effects of a dual orexin receptor antagonist called Lemborexant in adults aged 40 to 65 years who have insomnia. This Phase 2 randomized placebo-controlled cross-over study aims to determine whether Lemborexant can reduce blood levels of phosphorylated TAU181 and other biomarkers linked to neurodegeneration, compared to placebo. The study focuses on adults diagnosed with insomnia disorder as defined by DSM-5 criteria and with significant sleep difficulties and daytime impairments. Participants will receive 10 mg of Lemborexant orally each night for two weeks and will also take a matching placebo nightly for two weeks, separated by a 2 to 4 week washout period. After each treatment phase, participants will attend an overnight visit at the research facility lasting about 18 hours, which includes sleep time and various assessments such as high-density EEG, functional near-infrared spectroscopy, questionnaires, and pupillometry. Blood samples will be taken hourly for four hours after awakening during these overnight visits to measure biomarker levels. During the study, participants will complete a screening visit and three total visits including the overnight assessments. The research team will monitor blood biomarkers related to neurodegeneration, treatment effects, and safety throughout the study. The total participation duration includes two treatment periods, washout, and assessments, coordinated from research institutes in Sydney, Australia.

Researchers are evaluating the long-term safety and effects of nerandomilast in people with idiopathic pulmonary fibrosis (IPF) or progressive pulmonary fibrosis (PPF) who have previously completed treatment with nerandomilast in earlier studies. The study aims to understand how well participants tolerate nerandomilast over time, and whether it helps improve lung function, delays symptom worsening, reduces hospital visits, or impacts survival. This is a Phase 3 open-label extension trial. Participants take nerandomilast tablets daily for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study follows an open-label design where all participants receive nerandomilast. There are no placebo or comparator groups in this extension phase. Throughout the study, participants regularly visit their doctors for health assessments and lung function tests. Doctors monitor any health problems or side effects experienced during treatment. The main outcome measured is whether participants experience any adverse events up to the final follow-up visit, which occurs at week 99. This close monitoring helps evaluate the long-term safety and potential benefits of nerandomilast in this patient group.

Researchers are studying how changes in diet affect sleepiness, quality of life, and metabolic health in people with narcolepsy and idiopathic hypersomnia. The project focuses on comparing a 12-week whole-food ketogenic diet (WFKD) to a standard whole-food diet (WFD) to see if these well-researched dietary changes can improve symptoms and health outcomes in people living with these sleep disorders. This study also aims to explore patient preferences and factors affecting the wider use of these diets alongside usual care. Participants first follow a standard whole-food diet for three weeks, focusing on unprocessed or minimally processed foods. After this run-in period, they are randomly assigned to continue the standard diet or switch to a ketogenic diet with restricted carbohydrates aiming for nutritional ketosis for nine weeks. The ketogenic diet group will monitor blood and urine ketones to maintain target levels. Both diets are delivered with education about diet quality and strategies to overcome barriers to change. During the 12-week study, participants will be monitored for feasibility, tolerability, and compliance with the diet. Researchers will assess sleep-related outcomes such as daytime sleepiness, sleep onset latency, and psychomotor vigilance, along with metabolic health markers and quality of life measures. Data collection includes dietary monitoring, ketone testing, and questionnaires. The study also examines patient preferences for dietary versus medication treatments and gathers information on challenges in implementing these dietary changes.

This is a randomised open-label cross-over study of an inhaled formulation of melatonin (100 µg) versus oral melatonin tablets (4 mg) in adults with insomnia. The experiments performed for this trial will examine the effects of inhaled and oral melatonin on sleep microarchitecture such as sleep onset latency. To be enrolled in the trial, participants are required to complete an online pre-screening survey. Eligible participants will be directed to a separate webpage where they will be invited to review and download the Participant Information Sheet (PIS) and asked to provide their contact details and consent for a follow up call/email from the research team to book in a screening visit. At the screening visit, the study team will explain the study to each participant and provide the opportunity to ask any questions. The study team will also ensure participants have had ample time prior to the visit to read and understand the PIS. The consent form will be signed by both the participant and a medical officer and participants. Once participants have joined the efficacy study, they will be randomised into their first treatment group; inhaled melatonin (100 µg) or oral melatonin tablets (4 mg). Participants will attend the laboratory for an overnight visit then be treated with either inhaled melatonin or oral melatonin tablets before completing an overnight polysomnography sleep study. After the sleep study, participants will continue to take the study drug every night for two weeks and complete a sleep diary each morning to assess participant subjective perception of sleep quality. Once the two weeks of treatment have been completed, participants will visit the laboratory again to provide a blood sample that will be examined for biomarkers of neuroinflammation. There will be a 1 week washout period between treatment periods. The study will recruit primarily through social media advertisements. The study will be coordinated from the Woolcock Institute of Medical Research, Sydney, Macquarie University, NSW, 2113, Australia.

Researchers are studying adults aged 55 and older with insomnia to compare how an inhaled melatonin formulation behaves in the body versus oral melatonin tablets. The focus is to understand differences in how quickly melatonin reaches its peak level in the blood and how it is cleared from the body. This is an early phase 1, randomised, open-label crossover study designed to explore these pharmacokinetic differences in adults diagnosed with insomnia disorder. Participants will receive two types of melatonin treatments on separate visits: a 100 microgram dose delivered through an inhaler (two puffs) and a 4 mg oral dose (two tablets). Each treatment is taken once in the morning at a daytime laboratory visit. There is a one-week washout period between treatments. The inhaled melatonin is given via a pressurised metered dose inhaler, while the oral melatonin consists of tablets compliant with Australian quality standards. Participants will attend screening and two daytime visits where treatments are administered. Blood samples will be collected frequently for 8 hours after dosing to measure melatonin levels. Participants will also rate their sleepiness during blood collection using a standard scale. The study involves three visits total, including the screening, and is conducted at research institutes in Sydney, Australia. The main outcome measured is the time it takes for melatonin to reach its maximum concentration in the blood within 8 hours after treatment.

Researchers are investigating new treatment options for adults with advanced solid tumors that have a KRASG12C mutation. This mutation causes cancer cells to grow, and while the drug sotorasib is approved to target these cancer cells, it usually works only for a limited time before the cancer grows again. The study is testing BAY3498264, a drug designed to block a protein called SOS1 that works with KRAS, hoping this will enhance the effects of sotorasib by providing a longer or stronger response. This is a first-in-human Phase 1 study focusing on the safety and best dosing of BAY3498264 when combined with sotorasib. Participants will first take BAY3498264 alone for seven days, then receive it together with sotorasib in repeated 21-day cycles. Sotorasib is given daily at a standard approved dose alongside BAY3498264. Treatment will continue as long as it benefits participants without causing severe side effects, or until the cancer progresses, or if the participant or doctor decides to stop. The study consists of three parts: dose escalation, backfill, and expansion to determine safe dosing and to monitor effects. Throughout the study, participants will have blood and urine samples collected and undergo imaging scans such as CT, PET, MRI, and X-rays. Their heart health will be checked using ECGs. Researchers will closely monitor safety by tracking any side effects, serious adverse events, drug levels in the blood, and any toxicities during the first treatment cycle. The study will last approximately three years to assess the maximum tolerated dose and overall safety of the drug combination.

Researchers are evaluating insulin icodec, a once-weekly insulin injection, compared to insulin glargine, a once-daily injection, in adults with type 1 diabetes. The study aims to see how well weekly insulin icodec controls blood sugar levels compared to daily insulin glargine when both are combined with insulin aspart. This phase 3 study will last about 26 weeks, or roughly 8.5 months. Participants will receive either insulin icodec or insulin glargine, both given as subcutaneous injections. All participants will also use insulin aspart as a subcutaneous injection. The study compares these two insulin regimens to assess their effects on blood sugar control over the 26-week period. During the study, researchers will monitor changes in glycosylated hemoglobin (HbA1c) from the start of the study to week 26. Participants will follow the study protocol including self-measured plasma glucose profiles. Safety and efficacy will be evaluated throughout the treatment period to understand the impact of the insulin regimens on blood sugar control and participant health.

Researchers are evaluating FMC-376 in adults with advanced solid tumors that have a specific KRAS G12C mutation. This trial aims to assess the safety, pharmacokinetics, and clinical effects of FMC-376 in patients whose tumors are locally advanced, unresectable, or metastatic. The study is conducted in three parts: Phase 1A (dose escalation), Phase 1B (dose expansion), and Phase 2 (cohort expansion), focusing on multiple dose levels in this patient population. Participants will receive FMC-376 as an oral capsule taken daily. The study explores different dosing schedules across the phases to determine optimal dosing and further evaluate the treatment's effects. The study is open-label, meaning both researchers and participants know which treatment is being administered. During the study, participants will be closely monitored for adverse events and dose limiting toxicities up to 21 days, with safety assessments continuing for approximately 24 months. Researchers will also assess pharmacokinetics and clinical activity of FMC-376. Participants must meet certain health and function criteria before and during the study to ensure safety and reliable results.