Woolloongabba

Researchers are evaluating the safety and effectiveness of elenestinib BLU-263 combined with symptom directed therapy SDT compared to placebo plus SDT in adults with indolent systemic mastocytosis ISM whose symptoms are not well controlled by SDT alone. This randomized, double-blind, placebo-controlled Phase 23 study includes multiple parts to assess different doses and durations of elenestinib treatment, including an open-label extension for participants finishing earlier phases. The study also enrolls participants who have previously received an approved selective KIT inhibitor and includes pharmacokinetic groups. Participants receive oral elenestinib or placebo once daily alongside SDT, which is personalized based on individual symptom management needs. Part 1 focuses on short-term treatment lasting up to 12 weeks, while Part 2 extends treatment to approximately 48 weeks. Part 3 and other parts allow treatment for up to about 5 years. The study monitors participants through these phases to evaluate how elenestinib affects symptoms, disease markers, and safety over time. During the study, participants undergo regular assessments of symptoms using the ISM-Symptom Assessment Form ISM-SAF, laboratory tests including serum tryptase and KIT D816V allele levels, bone marrow evaluations, and quality of life measures. Researchers track adverse events and changes in disease-related factors at various points up to 5 years. This thorough monitoring helps measure treatment effects and safety over both short and long-term periods, with total participation lasting several years depending on the study part.

Researchers are evaluating ONM-501, a drug given as intratumoral injections, alone and in combination with cemiplimab, an immune checkpoint inhibitor, in patients with advanced solid tumors and lymphomas. This phase 1 study aims to find the maximum tolerated dose, minimum effective dose, and recommended dose for expansion of ONM-501. The study includes patients with various advanced cancers who have no alternative standard therapies available. The trial has three parts monotherapy dose escalation, combination therapy dose finding, and combination therapy dose expansion. ONM-501 is given once per week for three weeks followed by three weeks off, in 21-day cycles. Cemiplimab is given intravenously every three weeks during the combination phases. Dose escalation uses special methods to gradually increase doses, and after doses are established, patients will enroll in expansion cohorts for specific tumor types. Participants will have regular assessments including monitoring for side effects, blood tests to measure drug levels, and evaluation of tumor response over up to 24 months. Researchers will track treatment-emergent adverse events, dose-limiting toxicities, and serious adverse events. Outcomes such as objective response rate, duration of response, progression-free survival, and overall survival will also be recorded. The study involves close safety monitoring and follow-up throughout the treatment and observation periods.

Researchers are evaluating BGB-16673, an oral drug, in adults with various types of B-cell malignancies such as marginal zone lymphoma, follicular lymphoma, mantle cell lymphoma, chronic lymphocytic leukemia, Waldenstrm macroglobulinemia, diffuse large B-cell lymphoma, and Richters transformation. This study includes Phase 1 dose finding and safety expansion, followed by Phase 2 expansion cohorts to determine recommended doses and further assess safety and efficacy. The study is divided into several parts, starting with Phase 1 dose escalation to find safe dosage levels, including monotherapy dose escalation and safety expansion in selected doses. Phase 2 involves expansion cohorts where participants receive the recommended doses identified in Phase 1 for further safety and efficacy evaluation. Some cohorts include participants who have not received prior BTK inhibitors, and Japanese participants are also enrolled to assess safety. Treatments are orally administered. Participants will undergo regular assessments including monitoring for adverse events, disease response, and drug concentration levels in the blood at various time points. Researchers will measure outcomes such as overall response rate and progression-free survival over approximately three years. Safety and tolerability will be closely tracked, and quality of life questionnaires will be completed at scheduled intervals. Participation may last several years, including follow-up periods to monitor long-term effects.

Researchers are evaluating the safety and effectiveness of co-implanting the WiSE CRT System with an intracardiac pacemaker to achieve a completely leadless cardiac resynchronization therapy CRT for patients with heart failure. This single-arm, prospective, multicenter observational study focuses on delivering biventricular pacing by combining left ventricular pacing from the WiSE CRT System with right ventricular stimulation from a co-implanted device. The study aims to understand how well this leadless approach works and its related risks. Participants will receive the WiSE CRT System implanted to provide left ventricular pacing along with an intracardiac pacemaker for right ventricular pacing, together delivering biventricular stimulation. This therapy is given alongside guideline-directed medical therapy. The study observes participants over time without comparing to a control group. During the study, participants will be monitored for device- and procedure-related complications and for evidence of biventricular capture on electrocardiograms at one month and six months after implantation. Researchers will also assess changes in heart function, including ejection fraction, left ventricular end systolic volume, heart failure symptom class, and walking ability at six months. The study includes follow-up visits to evaluate safety and heart performance, with participation lasting at least six months.

Researchers are evaluating the safety, tolerability, and preliminary effectiveness of IMP1734, a PARP1 selective inhibitor, in people with advanced solid tumors. This study includes patients with breast cancer, metastatic prostate cancer, ovarian cancer, and other solid tumors who have previously received certain treatments. The goal is to find an optimal dose for future clinical development by studying how the drug affects the body and how the body processes it. The study has two parts Part 1 involves gradually increasing doses of IMP1734 given as a daily oral tablet to identify the highest safe dose or maximum achievable dose. This includes testing the drug alone and in combination with other treatments for specific cancers like metastatic prostate, ovarian, and breast cancer. Part 2 focuses on refining the dose to find the best amount for future studies. Treatment can last up to three years after the first dose. Participants will be monitored closely with assessments of side effects, blood tests to study drug levels and effects, and evaluations of tumor response using standard criteria. Safety monitoring continues up to 30 days after the last dose. Researchers measure how well the drug is tolerated and its impact on the cancer over time. The total participation may extend up to three years, with ongoing evaluations during this period.

Researchers are evaluating the efficacy and safety of rilvegostomig compared to pembrolizumab monotherapy as the first-line treatment for patients with metastatic non-small cell lung cancer mNSCLC whose tumors express high levels of PD-L1. This Phase III, randomized, double-blind, multicenter global study focuses on patients with mNSCLC without certain genetic mutations who are suitable for this treatment approach. Participants are randomly assigned to receive either rilvegostomig or pembrolizumab intravenously on Day 1 of each 21-day cycle. The study compares these two drugs over repeated treatment cycles as first-line therapy. Both treatments are biological agents given by infusion, and the study is designed to monitor their effects over up to approximately five years. During the trial, participants will undergo regular assessments including physical exams, imaging scans such as CT or MRI to measure tumor lesions, and laboratory tests to evaluate organ function. Researchers will closely monitor overall survival, progression-free survival, treatment response, duration of response, and patient-reported outcomes on physical functioning and quality of life. Safety and immunogenicity of rilvegostomig will also be evaluated. Participants are followed and assessed for up to five years to gather comprehensive data on treatment effects and long-term outcomes.

Frailty is a common condition in older adults that can increase the risk of poor health outcomes such as falls, hospital stays, and death. This research aims to study how assessing frailty before a colonoscopy might affect patient satisfaction and the number of colonoscopies avoided. The study is a prospective randomized controlled trial involving patients over 65 years old at Princess Alexandra Hospital, comparing a personalized approach using a frailty assessment to standard care. Participants will be randomly assigned to one of two groups. One group will receive a personalized approach that includes a frailty assessment using tests like grip strength, sit-to-stand timing, balance, and gait speed, along with questionnaires about medical conditions. The gastroenterologist will discuss the results and risks with the patient to help decide about having a surveillance colonoscopy. The other group will receive standard care, which involves discussing the benefits and risks of colonoscopy without the frailty assessment. During the study, participants will complete satisfaction questionnaires at the time of consultation and within weeks after any colonoscopy procedure. Researchers will track how many patients choose to avoid colonoscopy after receiving information and will monitor colorectal cancer-related complications and other causes of death within five years. The study will assess how the personalized approach may impact decision-making and patient satisfaction with outpatient services related to colonoscopy care.

Researchers are studying ZL-1503 in a phase 11b clinical trial to assess its safety, tolerability, and how the body processes the drug. The study includes both healthy adults and adults with moderate to severe atopic dermatitis AD, a skin condition characterized by inflammation and itching. This randomized, double-blind, placebo-controlled trial aims to better understand the effects of ZL-1503 in these groups. The trial has two parts Part A involves single increasing doses of ZL-1503 given to healthy volunteers, while Part B involves multiple increasing doses given to adults with moderate to severe AD. Participants will receive either ZL-1503 or a placebo. The study monitors participants closely during dosing to evaluate how the drug is tolerated and how it behaves in the body. Participants will undergo various assessments throughout the study, including monitoring for adverse events, vital signs, laboratory tests, and electrocardiograms ECGs for up to 48 weeks after the last dose. These evaluations help researchers track safety and any potential side effects. The total participation period can last up to 48 weeks following dosing to ensure thorough observation.

Researchers are evaluating the safety and effectiveness of three different dose regimens of MORF-057, a small molecule drug, in adults with moderately to severely active Crohns disease CD. This Phase 2, randomized, double-blind, placebo-controlled, multicenter study aims to compare these doses with a matching placebo during an induction treatment period. The study includes adult participants who have active symptoms of CD and have not adequately responded to other treatments. Participants will first undergo a 14-week induction period where they receive either one of the three blinded MORF-057 dose regimens or a matching placebo, all taken orally. Following this, all participants enter a 38-week maintenance period receiving open-label MORF-057. Those who complete this 52-week treatment phase may have the chance to continue treatment for an additional 52 weeks during a long-term extension. MORF-057 is designed to selectively inhibit integrin 47. During the study, participants will have their disease activity monitored using endoscopic assessments and clinical symptom scores, such as the Simple Endoscopic Score for Crohns Disease SES-CD and the Crohns Disease Activity Index CDAI. Researchers will assess the proportion of participants showing endoscopic response and clinical remission at Week 14. Safety and adherence will be closely followed throughout the treatment and extension phases. The entire study spans up to 6 years, allowing for long-term evaluation.

Researchers are evaluating the safety and effects of a medicine called fosmanogepix for treating candidemia and invasive candidiasis, which are serious fungal infections caused by Candida yeast. This Phase 3 clinical trial compares fosmanogepix to the standard treatment using caspofungin followed by fluconazole, aiming to show that fosmanogepix is not worse than the standard treatment by a margin of 15%. The study includes adult patients diagnosed with these infections and is sponsored by Basilea Pharmaceutica. Participants are randomly assigned to one of two groups two-thirds receive fosmanogepix intravenously, with an option to switch to oral tablets, while one-third receive caspofungin intravenously followed by oral fluconazole. Matching placebos are given to maintain blinding. Treatments are given daily, first by IV infusion at the clinic and then orally either at the clinic or at home if discharged. Treatment duration can be up to six weeks, depending on infection clearance and symptom improvement. Participants will be monitored through multiple study visits, with assessments including survival status at 30 days, treatment success at the end of treatment, and follow-up evaluations six weeks after stopping treatment. Additional evaluations include clinical and mycological responses, blood cultures, safety monitoring such as adverse events, lab tests, neurological exams, ECGs, and drug concentration measurements. The total study duration for each participant may be approximately 12.5 weeks, considering treatment and follow-up periods.