ANZCTR search results

ASPREE-XT is a post-treatment, longitudinal observational follow-up study of ASPREE participants. Although the ASPREE trial medication was ceased, the study activity was not stopped and ASPREE participants are continuing with scheduled visits and phone calls. An observational follow-up phase (ASPREE-XT), began in January, 2018. This will enable the monitoring of possible delayed effects of aspirin treatment, primarily on cancer incidence, metastases and mortality. In addition to monitoring the incidence of malignancy within the ASPREE cohort, the opportunity will be taken to observe any other residual effects of aspirin on the endpoints being monitored in the cohort. Continuity of contact with study participants is the key to retention of the cohort for any ongoing or future studies.

Blood-based ADRD biomarkers show tremendous promise as a non-invasive method to predict and diagnose ADRD. We will measure changes in these biomarkers to determine whether their ability to predict ADRD differs by sex or by other characteristics, and develop a sex-specific ADRD risk score for personalized medicine and clinical trials.

This study is a Phase 3, non-randomized, multicenter, efficacy and safety study in adult patients with Gaucher disease Type 1, on stable treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study aims to confirm the efficacy and safety of FLT201 in this population after discontinuation of ERT/SRT.

The current standard of care treatment for adult patients with acute myeloid leukemia (AML) consists of chemotherapy and, if indicated, donor stem cell transplantation. Bleximenib blocks the interaction between a protein called menin and another protein called KMT2A in the leukemia cells. When this interaction is disrupted in AML with mutations in the NPM1 or KMT2A gene, bleximenib can cause leukemia cells to die. The main objective is to assess if treatment with bleximenib, when added to chemotherapy treatment will improve treatment outcome in adult participants with newly diagnosed AML who present with mutations in the NPM1 or KMT2A genes. This is a randomized, double-blind, placebo-controlled, phase 3 clinical trial. All of the participants will receive standard chemotherapy treatment, combined with either bleximenib or a placebo. A placebo is a substance that looks like the study medicine but has no active ingredients (e.g., a sugar pill). In a double blind trial neither the participant nor the doctor know if placebo or active study drug is given. After the end of the protocol treatment there will be an observational follow-up of 4 years from the time of inclusion of the last patient. The results of the different treatment groups will be compared. 875 previously untreated patients with AML with a specific change in the DNA of the leukemia cells (a KMT2A rearrangement or a NPM1 mutation) will be included. Participants must be 18 years or older and considered eligible for intensive chemotherapy.

Study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD), and effects on low-density lipoprotein cholesterol (LDL-C) and triglycerides (TGs) of single-dose ARO-DIMERPA and multiple doses of ARO-DIMERPA in adult participants with mixed hyperlipidemia.

The purpose of this study is to evaluate the effect and safety of orforglipron once daily in participants with Fontaine II peripheral arterial disease (PAD). Participation in the study will last about 58 weeks.

This is a retrospective, observational study looking at real-world prescription data in Australia. The study focuses on adult men with metastatic hormone-sensitive prostate cancer (mHSPC), a type of prostate cancer that has spread but still responds to hormone therapy. The main goal is to understand the treatment patterns, characteristics, and outcomes for patients who are receiving a class of drugs called Androgen Receptor Pathway Inhibitors (ARPIs), such as darolutamide, enzalutamide, apalutamide, or abiraterone. These drugs are often used in combination with standard Androgen Deprivation Therapy (ADT). The research will use two large, de-identified Australian prescription databases: the Pharmaceutical Benefits Scheme (PBS) and the NostraData retail pharmacy dataset. By analyzing this information, the study aims to describe how these treatments are being used over time, including what proportion of patients receive doublet therapy (ARPI + ADT) or triplet therapy (ARPI + ADT + docetaxel chemotherapy). It will also describe the characteristics of the patients receiving these therapies, such as their age and location. Secondary goals include understanding how well patients adhere to their prescribed ARPI treatment and tracking any changes in medication dosage over time. Since the study uses existing, anonymized data, there is no direct contact with patients and individual patient consent is not required. The findings will provide valuable real-world insights into the use of ARPIs in Australia, which can help inform clinical practice and improve care for men with mHSPC.

The purpose of this study is to: * Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed * Demonstrate superiority of nucresiran compared to in-study vutrisiran with respect to serum transthyretin (TTR) levels

This clinical trial is an open-label, single-arm, non-randomized, dose-escalation and dose-expansion study targeting subjects with unresectable, advanced, malignant solid tumors who have failed or are unsuitable for standard treatments or refused the existing treatments. This study is divided into a dose-escalation phase (Phase I) and a dose-expansion phase (Phase II). Phase I (dose escalation) is designed to preliminarily evaluate the safety and tolerability of VIB305 in advanced solid tumors, to determine the nature and incidence of dose-limiting toxicities (DLTs), and thereby to identify the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D). Based on the findings from the Phase I portion for evaluation in the Phase II portion. Phase II (dose expansion) will enroll additional cohorts to further assess the safety and tolerability, PK profile, preliminary antitumor activity and immunogenicity of VIB305 in specific tumor types (selected based on all available data).

The purpose of this study is to learn more about a new medicine called PF-08634404, and how well it works in people with cancer of the colon or rectum (CRC)). The goal is to understand if the new study medicine, combined with chemotherapy that is approved for colorectal cancer, can help people whose cancer has spread or returned after treatments taken before. To join the study, participants must meet the following conditions: * Be 18 years or older. * Have colorectal cancer that has spread to other parts of your body. * Be in good enough health to receive study treatment. * Should not be pregnant before starting treatment. Participants will be randomized (like flipping a coin) to one of 2 different treatment arms. The first arm (Arm A) will include the new medicine PF-08634404 in combination with chemotherapy that is approved for colorectal cancer, and the second arm (Arm B) will include an approved medicine for colorectal cancer, called Bevacizumab, in combination with chemotherapy that is approved for this type of cancer. Participants and their doctors will not know which arm they are being assigned to. Participants will receive all the study medications through intravenous (IV) infusions, which means the medicine is given directly into a vein. The treatment will be given in cycles, and participants may continue receiving it if it is helping and they are not experiencing serious side effects. The medicine will be given at a clinical site, where trained medical staff will check participants during and after each treatment. * The study is expected to last approximately 33 months for each participant. * Participants will have regular visits to the study site for treatment, health checks, and tests. * After stopping treatment, participants will return for a final visit about 30 to37 days later to check their health and review any side effects. * Follow-up will continue every 12 weeks by phone or in person or by reviewing health records to check on health status and any new treatments.