Westmead

Researchers are conducting a prospective multicenter, open-label, dose-escalation trial to evaluate the safety, tolerability, and pharmacodynamics of 4D-310 in adults with Fabry Disease who have cardiac involvement. The study includes both males and females aged 18 to 65 years. Fabry Disease is a genetic condition, and this trial focuses on participants with a confirmed pathogenic GLA mutation and cardiac symptoms related to the disease. Participants will receive a single intravenous (IV) dose of the gene therapy 4D-310. The trial is designed to carefully escalate the dose to monitor effects and safety. This study does not involve repeated dosing but focuses on the effects following one administration of the therapy. During the study, participants will be monitored for adverse events over the course of one year to assess both the incidence and severity of any side effects. Other evaluations will include safety assessments and pharmacodynamic measurements to understand how the therapy affects the body. The total involvement period includes this year-long follow-up to gather comprehensive safety data.

Researchers are studying whether calderasib alone or combined with cetuximab can treat advanced solid tumors in people who have the KRAS G12C mutation. This phase 2, open-label trial aims to find out how many participants respond to these treatments and to compare their safety and tolerability. Participants receive calderasib by mouth and cetuximab through intravenous infusion. The study includes people with locally advanced or metastatic solid tumors other than colorectal cancer, who have already undergone standard treatments. The trial monitors response and side effects over time as participants receive either calderasib alone or in combination with cetuximab. During the study, participants undergo regular assessments to measure tumor response and track any side effects or adverse events. Researchers record how many people experience treatment-related side effects and how many stop treatment due to these effects. The study follows participants for up to approximately 76 months to assess long-term outcomes and safety.

Researchers are investigating sacituzumab tirumotecan (MK-2870) alone or combined with other treatments to treat certain gastrointestinal cancers. These include colorectal cancer that cannot be removed by surgery or has spread, advanced pancreatic ductal adenocarcinoma, and biliary tract cancer. The study aims to understand the safety and tolerability of sacituzumab tirumotecan and measure how many participants respond to the treatment by having their cancer shrink or disappear. Participants may receive sacituzumab tirumotecan by intravenous infusion alone or with other anticancer drugs such as fluorouracil (5-FU), leucovorin or levoleucovorin, cisplatin, and pembrolizumab. Rescue medications like diphenhydramine, H2 receptor antagonists, acetaminophen, dexamethasone, and a steroid mouthwash are given to prevent infusion reactions and oral side effects. Supportive care treatments for side effects, including antidiarrheal and antiemetic agents, are allowed throughout the study. During the study, researchers monitor participants for dose-limiting toxicities within about 4 weeks and track adverse events, treatment discontinuations, and tumor response over up to approximately 63 months. Assessments include safety evaluations and measuring cancer response using standardized criteria. This long-term follow-up helps evaluate both the effectiveness and safety of the treatments being studied.

Researchers are evaluating LY3884961, a genetic therapy using a non-replicating viral vector, in adults with peripheral symptoms of Gaucher Disease, a rare genetic disorder. This Phase 1/2 open-label study aims to find safe dose levels and assess the treatment's safety and tolerability over a long period. The study includes patients with confirmed GBA1 gene variants who have been on stable treatment for at least two years. Participants will receive a single intravenous dose of LY3884961. The study starts with three dose-finding groups, each including three patients, followed by an expansion group with up to six patients. After dosing, the first 18 months focus on monitoring safety, immune response, biomarkers, and signs of treatment effect. Then, participants will be followed for an additional 42 months to continue safety and efficacy evaluations. Each participant's involvement lasts about five years, including up to 60 days for screening before treatment. Throughout the study, researchers will conduct medical assessments, laboratory tests, and biomarker analyses. They will closely monitor for any adverse events and immune reactions. The main goal is to track the occurrence and severity of any side effects that develop during the entire five-year period.

Researchers are evaluating a combination treatment using BNT326 and BNT327 in adults with advanced or metastatic non-small cell lung cancer (NSCLC), including those with relapsed, progressive, or treatment-nafve disease. This multi-site, open-label study includes dose-finding and dose-expansion phases to investigate the safety, tolerability, and preliminary effectiveness of this combination therapy. The study targets patients whose tumors are advanced, metastatic, or recurrent with no curative treatment options available and includes participants with different genomic alterations. The study is divided into several parts: Part 1 is a dose escalation phase to find safe dose levels of BNT326 with BNT327; Part 2a expands the dose to further evaluate safety and initial efficacy; Part 2b focuses on dose optimization and understanding the contributions of each component. Participants receive intravenous infusions of BNT326 and BNT327, with some cohorts possibly receiving additional treatments such as pembrolizumab or standard chemotherapy. Treatment continues until disease progression, unacceptable side effects, withdrawal, or a maximum of 24 months. Dose levels for certain cohorts are determined based on earlier phase data, and some parts include randomization to different treatment groups. Participants undergo a screening period before starting treatment, followed by treatment, safety follow-up, efficacy follow-up, and long-term survival monitoring, totaling about 36 months. Researchers assess dose-limiting toxicities within the first 21 days of treatment and monitor adverse events, treatment interruptions, and objective response rates up to 36 months. Tumor measurements, safety labs, imaging, and patient health status are regularly evaluated. The study tracks tolerability and efficacy while ensuring participant safety throughout treatment and follow-up.

Researchers are evaluating the safety of aerosolized RSP-1502 in people with cystic fibrosis who have chronic lung infections caused by Pseudomonas aeruginosa. This phase 1b/2a study compares different doses of RSP-1502 to an active control, aiming to find the maximum tolerated dose (MTD) and assess safety outcomes. Participants must meet specific lung function and infection criteria to join the study. The study involves administering RSP-1502 or an active control solution by inhalation using a nebulizer for 14 days. RSP-1502 contains tobramycin and CaEDTA in a sterile solution, while the active control is a tobramycin inhalation solution. After dose escalation to identify the MTD, a dose expansion phase compares the MTD of RSP-1502 to the active control for another 14 days. Participants will then be followed for 14 days after treatment ends. Participants will have their lung function tested with spirometry and undergo electrocardiograms on specific days during treatment. Researchers will monitor for any treatment-related adverse events and serious adverse events throughout the 28-day treatment and follow-up period. They will also track pulmonary exacerbations and other safety measures. The total participation includes dosing and a 14-day follow-up after treatment completion.

Researchers are conducting a Phase 1/2 study to evaluate the safety and tolerability of BDC-4182, an immune stimulating antibody conjugate, in patients with advanced gastric and gastroesophageal cancers. This first-in-human trial aims to find the recommended Phase 2 dose by gradually increasing doses and monitoring participants closely. The study focuses on patients with measurable metastatic or unresectable cancer who have already undergone prior standard treatments or cannot tolerate them. Participants will receive BDC-4182, which combines an anti-claudin 18.2 monoclonal antibody with a TLR 7/8 dual agonist, delivered as a single agent. The study includes a dose escalation phase where participants are enrolled in cohorts until the maximum tolerated dose is found. Additional participants may join backfill cohorts for further safety data or the expansion phase at the recommended dose level. Throughout the study, participants will undergo biopsies or tumor sample collection before enrollment. Researchers will monitor adverse events up to around two years, including serious and dose-limiting toxicities within the first 21 days. Organ function and other health assessments will be performed to ensure safety. The trial carefully tracks treatment effects and tolerability in this patient population over the course of the study.

Primary immune thrombocytopenia (ITP) is a condition in which the immune system mistakenly destroys platelets, the cells that help stop bleeding. This leads to a low platelet count, making it easier to bruise or bleed. The trial investigates the long-term safety, tolerability, and effectiveness of mezagitamab in adults with chronic primary ITP who have previously participated in certain mezagitamab studies. It also examines how the body processes mezagitamab over time. Participants who completed the previous mezagitamab studies TAK-079-3002 or TAK-079-1004 and meet specific criteria will receive mezagitamab as a subcutaneous injection during this continuation study. The study is open-label and multicenter, focusing on continued treatment based on protocol requirements. The medication is given under medical supervision, and participants return to the study clinic several times throughout the study. During their participation, individuals will undergo regular assessments including monitoring for treatment-emergent adverse events and serious adverse events up to approximately 108 weeks. Researchers will track safety by noting any adverse events that lead to permanent withdrawal from mezagitamab. The study includes physical evaluations, laboratory tests, and ongoing safety monitoring to understand how well participants tolerate the treatment and how effective it is over the long term.

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.

The purpose of this study is to assess the long-term safety and tolerability after an intravitreal injection (a shot of medicine into the eye) of JNJ-81201887 administered in parent clinical studies.