Perth

Researchers are evaluating the safety, tolerability, and pharmacodynamics of 4D-310, a gene therapy, in adults with Fabry Disease who have heart involvement. This open-label, dose-escalation trial includes adult males and females aged 18 to 65 years and aims to understand how the treatment works after a single intravenous dose. Fabry Disease is a condition that affects multiple organs, including the heart, and requires new treatment approaches. Participants receive one single intravenous administration of 4D-310 at different dose levels as part of the study. The trial includes several dose groups, with some dose levels no longer enrolling. The study is conducted across multiple centers and is designed to carefully monitor responses to the gene therapy over time. During the study, participants will be regularly assessed for safety by tracking any adverse events for one year following treatment. Researchers will also monitor how the body responds to the therapy through various evaluations. The total participation time includes screening, treatment, and follow-up visits to ensure thorough observation of treatment effects and safety.

Researchers are studying new treatment options for people with high-risk non-muscle invasive bladder cancer HR NMIBC, including cases with carcinoma in situ CIS. HR NMIBC affects the lining of the bladder but has not spread to muscle or beyond. The study aims to learn if adding intismeran autogene V940 to the standard Bacillus Calmette-Guerin BCG immunotherapy can improve outcomes by helping the immune system attack the cancer more effectively. Participants are divided into groups receiving different treatments. One group Cohort A receives both intismeran autogene via intramuscular injection every 3 weeks for 9 doses and BCG instillations weekly in specific weeks over about 75 weeks. Another group receives only BCG following the same weekly schedule. A third group Cohort B receives intismeran autogene alone every 3 weeks for 9 doses. The study evaluates these treatments over several years. During the study, participants will have regular treatments and follow-up visits where researchers will monitor cancer progression, recurrence, and survival for up to approximately 5 years. Assessments include event-free survival, recurrence-free survival, overall survival, response rates, time to cystectomy, and safety outcomes such as adverse events and treatment discontinuation. The study is randomized and open-label, with detailed long-term monitoring planned.

Researchers are investigating new medicines for children and young people up to 25 years old with relapsed or refractory B-cell non-Hodgkin Lymphoma B-NHL, a type of cancer affecting lymph nodes and organs like the liver or spleen. This international adaptive trial aims to find safer and more effective treatments, focusing on three groups receiving different novel therapies. The study uses a design that allows adding or removing treatments based on their effectiveness and safety in this rare cancer. Participants will receive one of three treatments odronextamab given by intravenous infusion weekly and then less frequently over up to two years loncastuximab tesirine combined with modified R-ICE chemotherapy for up to three cycles or CAR T-cell therapy with details to be confirmed. These treatments are tested in parallel groups, and if a medicine appears ineffective, it may be stopped and replaced by another. The trial allows children to switch groups if their cancer does not respond. During the study, researchers will monitor participants through regular assessments including imaging and laboratory tests to evaluate cancer response and side effects. They will check treatment responses at specific times during treatment cycles and follow patients for at least two years after treatment to monitor long-term outcomes and safety. This includes tracking survival times, adverse events, and overall treatment effectiveness to provide important information about these new therapies.

This research aims to gather long-term safety and effectiveness information for people treated with ibrutinib, a medicine taken by mouth that blocks a specific enzyme called brutons tyrosine kinase. The study focuses on participants who previously took part in ibrutinib studies that have finished and are still receiving ibrutinib treatment, continuing to benefit from it. It is an open-label study, meaning both participants and researchers know the treatment being given. Participants will continue taking ibrutinib capsules daily at the dose they were given in their prior study until the doctor decides the treatment is no longer helpful due to disease progression or side effects, the participant chooses to stop, other treatment options become available, or the study ends. Safety will be monitored throughout, and effectiveness data may be combined with previous study results. No formal testing of hypotheses is planned in this extension. During the study, participants will be regularly monitored for safety and disease status. The main outcome is the number of participants experiencing side effects within 30 days after the last ibrutinib dose or before starting another cancer therapy. Participants may continue treatment until alternative access to ibrutinib is arranged or the study ends, which is planned for December 2029. Researchers will collect ongoing data to understand the long-term effects of ibrutinib treatment.

Researchers are studying chronic post-surgical pain CPSP in children aged 0 to 16 years who undergo common pediatric surgeries such as laparoscopic appendicectomy, scrotal exploration, orchidopexy, hypospadias repair, and circumcision. The study aims to understand how often CPSP occurs, its risk factors, and its effects on childrens physical, emotional, and social well-being. This observational study will collect information through questionnaires from before surgery up to one year after surgery. Participants will be grouped based on the type of surgery they receive elective surgeries including circumcision, hypospadias repair, orchidopexy, or laparoscopic appendicectomy, and emergency surgeries including laparoscopic appendicectomy or scrotal exploration. The study observes these groups without providing treatments, focusing on collecting data about their pain experiences and related factors over time. Children and their families will complete questionnaires at six different times before surgery, on Day 2 after surgery, at 3 to 4 weeks, 3 to 4 months, and 10 to 12 months post-surgery. These questionnaires assess pain levels, function, anxiety, and other factors. Researchers will track the prevalence of chronic post-surgical pain at 10 to 12 months and identify potential risk factors to help improve future care and guidelines for children at risk of long-term pain.

Researchers are conducting a Phase 1 clinical trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of BW-41012 when given as a single subcutaneous injection to healthy adults aged 18 to 65 years. This randomized, double-blind, placebo-controlled study includes approximately 40 men and women divided into 5 cohorts to assess different dose levels of BW-41012. Participants will receive a single ascending dose of BW-41012 or placebo via subcutaneous injection. The study is structured into 5 cohorts, each with 8 participants receiving either the investigational drug or placebo. The trial employs a quadruple blinding and parallel assignment model to evaluate the investigational drugs effects compared to placebo. During the study, participants will undergo monitoring for up to 24 hours to assess primary endpoints, with additional evaluations extending up to 8 days and long-term safety follow-up up to 360 days. Assessments include laboratory tests, clinical evaluations, and safety monitoring to track drug effects and tolerability. The total participation duration can last up to nearly one year to ensure comprehensive evaluation of BW-41012.

Researchers are investigating the drug OKN4395, alone and combined with pembrolizumab, in adults with advanced solid tumors. This Phase 1 study aims to assess the safety, tolerability, blood levels, and anti-tumor activity of OKN4395 both as a single treatment and alongside pembrolizumab. The study focuses on tumors with a COX2-associated immunosuppressive pathway and includes multiple cancer types such as sarcoma, non-small cell lung cancer, colorectal cancer, and gastric cancer. The study is divided into two main parts. Part 1a includes dose escalation of OKN4395 alone or with pembrolizumab every 21 days, increasing doses based on safety evaluations, and a substudy testing how food and stomach acid affect OKN4395 blood levels. Part 1b evaluates OKN4395 alone or combined with pembrolizumab in four cancer cohorts. Participants receive oral OKN4395 twice daily, with pembrolizumab given intravenously every three weeks where applicable. The substudy involves dosing under fasting, fed, and high stomach pH conditions using famotidine. Participants will be monitored through regular visits lasting up to 27 months for Part 1a and up to 12 months for Part 1b. Assessments include safety checks for side effects, blood tests for drug levels and lab abnormalities, ECGs, tumor measurements, and evaluation of treatment response and progression. The study tracks dose adjustments, adverse events, and survival outcomes to understand the drugs effects and tolerability in solid tumor patients.

Researchers are evaluating the safety, tolerability, how the body processes pharmacokinetics, how the body responds pharmacodynamics, and effectiveness of TERN-701, a selective inhibitor targeting BCR-ABL1, in adults with chronic phase chronic myeloid leukemia who have received prior treatment. This Phase 12 trial focuses on participants with treatment challenges such as resistance or intolerance to previous therapies. The study is sponsored by Terns, Inc. and aims to better understand TERN-701s impact in this patient group. The trial has two main parts. Part 1 involves dose escalation where participants receive increasing doses of TERN-701 once daily to determine safe and effective dose levels. Part 2 includes randomized dose expansion cohorts to further assess safety and efficacy at two selected doses from Part 1, and an additional mutation cohort Part 2m evaluating a specific 500 mg dose in participants with certain resistance mutations. Treatment is given continuously in 28-day cycles with scheduled clinic visits at specified days during the first cycle and then on Day 1 of subsequent cycles. Participants will be closely monitored through regular visits for safety, tolerability, and response to treatment. Researchers will evaluate dose-limiting toxicities, adverse events, hematologic and molecular responses, and changes in BCR-ABL1 transcript levels over up to three years. Approximately 180 participants are expected to enroll, contributing to comprehensive data collection on TERN-701s performance and safety in this chronic leukemia population.

Researchers are studying bleximenib, an oral drug, in participants with acute leukemia to find the best dose and evaluate its safety and effectiveness. The study includes Phase 1 dose escalation to find recommended doses and Phase 1 dose expansion and Phase 2 to assess safety, tolerability, and anti-leukemia activity. Participants include both pediatric and adult patients with relapsed or refractory acute leukemia, especially those with specific genetic alterations. In Phase 1 Part 1, participants receive increasing doses of bleximenib orally to identify recommended doses based on tolerance. In Phase 1 Part 2, participants receive bleximenib at these doses to further evaluate safety. Phase 2 participants take the recommended dose to study the drugs effect on leukemia. The study monitors participants up to 4 years and 9 months for safety and treatment response. Participants will undergo assessments including monitoring adverse events, dose-limiting toxicities, and treatment responses. Blood tests will measure drug levels and leukemia remission rates. Safety and efficacy are tracked throughout treatment and follow-up, with a focus on remission rates and survival outcomes. The total study duration extends to September 2030, allowing long-term evaluation of bleximenib.

Researchers are studying sonrotoclax alone and in combination with dexamethasone plus carfilzomib, daratumumab, or pomalidomide to evaluate its safety, tolerability, and effectiveness in adults with relapsed or refractory multiple myeloma who have the chromosomal translocation t1114. This phase 1b2 trial aims to find the best doses and assess how well these treatments work, focusing on patients whose disease has progressed after previous therapies. Participants receive sonrotoclax as an oral daily medication, with dexamethasone given once weekly either orally or intravenously. Additional drugs include carfilzomib administered intravenously weekly, daratumumab given subcutaneously weekly, and pomalidomide taken orally daily. The study includes a dose-escalation phase to identify the maximum tolerated dose and up to seven expansion groups to further evaluate safety and response in different treatment combinations. During the study, participants are closely monitored for side effects and treatment responses through regular assessments including blood tests and bone marrow analysis. Key outcomes measured include dose-limiting toxicities, adverse events, and various levels of treatment response over approximately four years. This comprehensive follow-up helps researchers understand how the treatments affect disease progression and overall survival.