ANZCTR search results

This Phase 1, multi-center, open-label, dose escalation and dose optimization study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PDx), and preliminary clinical activity of MOMA-341 administered orally as a single agent or combination therapy in patients with microsatellite instability high (MSI-H) or DNA mismatch repair deficiency (dMMR) solid tumors.

This is a first-in-human clinical study of PEP08, a novel cancer therapy being evaluated both as monotherapy and in combination with other treatments in patients with advanced or metastatic solid tumors harboring MTAP deletion. The study will be conducted in three parts, with Part 1 currently open for enrollment. The primary objectives of the study are to: * Evaluate the safety and tolerability of PEP08, PK and PD * Determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) * Assess preliminary signs of anti-tumor activity of PEP08 Key study questions include: * What is the recommended dose of PEP08 for further development? * Wht is the tolerable dose of PEP08 when administered alone or in combination? * Does PEP08 show early evidence of clinical activity in patients with MTAP-deleted tumors? Participants in the study will: * Receive PEP08 alone or in combination with another anti-cancer agent, depending on the study part * Attend regular clinic visits for treatment administration, laboratory assessments, and tumor evaluations * Be enrolled in one of the following study phases over time: * \- Part 1: Monotherapy dose escalation (currently enrolling). * \- Parts 2 and 3 (monotherapy extension and combination therapy) will be activated in future protocol amendments.

The purpose of this study is to evaluate the efficacy and safety of BGB-16673 alone compared with pirtobrutinib in patients with relapsed or refractory (R/R) chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who had been previously treated with a covalent Bruton tyrosine kinase inhibitor (cBTKi).

This study will examine how the immune system responds to a flu virus (H3N2) during and after infection in health adults aged between 18 and 50 while in an inpatient facility. The study uses a specific flu virus called the H3N2 influenza challenge virus, that was produced specifically for use in clinical research in controlled conditions. From a previous study, mild to moderate symptoms are expected. This is the first time that a flu challenge study has been undertaken in Australia.

The purpose of this global, multicenter, open-label, Phase 4 clinical extension study is to evaluate the long-term safety and efficacy of revakinagene taroretcel-lwey (Encelto™; hereinafter referred to as NT-501), in participants with macular telangiectasia type 2 (MacTel) who previously received the intraocular implant in a Phase 1, Phase 2, or Phase 3 clinical study. In addition, this study will evaluate the safety and efficacy of NT501 in participants who previously underwent the sham procedure in a Phase 3 MacTel clinical study and elect to have NT-501 implanted intraocularly in this Phase 4 study.

The goal of this Phase 3, randomized study is to assess the safety, efficacy, tolerability, and pharmacokinetics (PK) of oral JNT-517 in adults (18 years of age or older) with PKU. Participants will receive either JNT-517 or placebo and will be blinded to their treatment assignment. Participants will have a 2 in 3 (or approximately 67%) chance of receiving JNT-517 during the first part of the study which will last approximately six weeks. During the second part of the study every participant who continues in the study will receive one of two doses of JNT-517 for an additional 46 weeks. The study requires a screening period of up to 35 days to ensure dietary stabilization and amino acid levels required to meet study eligibility. In total, participation in the study could last for up to 400 days. Participants will: Take 75 mg JNT-517 or 150 mg JNT-517, or a placebo BID (2x per day) for approximately 365 days; Visit the clinic or have a mobile health nurse visit your home for checkups and tests; Collect urine sample at home and bring to clinic on specified days; Keep a food diary 3 days before each study visit

The aim for this study is to investigate the ability of 64Cu-SAR-bisPSMA PET/CT to detect recurrence of prostate cancer

This Phase I, randomized, double-blind, parallel-controlled study is to be conducted in healthy adults aged 60 and above. Participants will be randomly assigned in a 1:1 ratio to receive either 26-valent Pneumococcal Conjugate Vaccine (PCV26) or the comparator (PCV13) on Day 1 (Visit 1). Solicited adverse events (AEs) will be collected for 7 days post-vaccination and unsolicited AEs for 28 days post-vaccination, with safety data limited to serious adverse events (SAEs), and newly diagnosed chronic medical conditions (NDCMCs) collected up to 6 months post-vaccination.

This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. Approximately 21 participants from across UK and Australia are expected to be enrolled, depending on safety review committee (SRC) throughout the course of the study. Participants may be assigned to any of the following: 1. A single 60ug dose of PYC-001 2. Three doses of 10ug PYC-001 at an interval of 8 weeks 3. Three doses of 10ug PYC-001 at an interval of 12 weeks 4. Three doses of 30ug PYC-001 at an interval of 8 weeks 5. Three doses of 30ug PYC-001 at an interval of 12 weeks Following completion of the 4 week safety review of the single 60ug of PYC-001 cohort, and if the 60 µg dose level is deemed safe by the SRC, the following cohorts will also be available: 6. Three doses of 60ug PYC-001 at an interval of 8 weeks 7. Three doses of 60ug PYC-001 at an interval of 12 weeks

Rationale: LTI-03 is an experimental medication breathed into the lungs using an inhaler. It is being studied for the treatment of Idiopathic Pulmonary Fibrosis (IPF). IPF is a progressive, fatal lung disease caused by the death of lung cells involved in oxygen uptake and by progressive fibrosis (scarring) of the lungs. As the disease progresses, patients experience loss of lung function and increased breathing problems. LTI-03 is hypothesized to treat IPF by protecting and restoring the function of the oxygen uptake cells and by controlling lung fibrosis which may result in improving lung scarring. The purpose of this research is to evaluate LTI-03 including: its safety, whether it causes side effects, whether it improves lung scarring, and whether it improves IPF symptoms. LTI-03 will be compared to placebo in patients diagnosed with IPF within the last 5 years. Patients on a stable dose of nintedanib, pirfenidone, or nerandomilast (if available by prescription) may participate. Trial Design: This is a Phase 2, randomized, double-blind, placebo-controlled, multi-center study that includes a 28-day Screening Period, a 24-week Treatment Period, and 4-week Follow-up Period. Study Assessments: Up to 9 visits to the study clinic will be required. Safety and tolerability will be evaluated with the following assessments: physical examination; collection of vital sign data (heart rate, blood pressure, respiratory rate and peripheral oxygen saturation \[SpO2\] via pulse oximetry); heart data collected by 12-lead electrocardiogram; and collection of blood samples for safety laboratory tests. In addition, participants will be asked about any adverse events (side effects) they have experienced between clinic visits, if they have changed any medications, and if they are able to properly use their study drug inhaler. Participants will undergo a lung function test (spirometry) at every visit, which will be used to evaluate both safety and efficacy. Another test measuring the diffusion capacity of the lungs for carbon monoxide (DLCO) will be required at Screening only. Blood samples will also be collected at each visit to measure disease biomarkers. At select visits patients will be asked to complete the Living with Pulmonary Fibrosis questionnaire to evaluate their IPF symptoms. Participants will also undergo a specialized lung scan (HRCT) at Baseline and at the End of Treatment to measure changes in lung fibrosis. Interventions: LTI-03 and placebo are provided in powder-filled capsules that participants will self- administer using an inhaler. Placebo capsules look like LTI-03 capsules but have no active ingredients. Approximately 120 participants will be randomly assigned in a blinded manner to one of study drug treatment groups.