East Melbourne

Researchers are investigating whether sacituzumab tirumotecan alone or combined with other treatments can treat certain advanced or unresectable gastrointestinal cancers, including colorectal cancer, pancreatic ductal adenocarcinoma, and biliary tract cancer. The study aims to understand the safety and tolerability of sacituzumab tirumotecan and how well the cancer responds to these treatments. Participants will receive sacituzumab tirumotecan in different dose levels either combined with chemotherapy every two weeks in a 4-week cycle, alone every two weeks in a 4-week cycle, or combined with cisplatin and pembrolizumab in a 3-week cycle. Treatment continues until the cancer worsens or participants cannot tolerate it. Cisplatin is given up to approximately six months, and pembrolizumab is administered for up to about two years in the combination group. During the study, participants will have regular assessments to monitor safety, side effects, and how the cancer responds via imaging and clinical evaluation. Researchers will track dose-limiting toxicities, adverse events, treatment discontinuations due to side effects, and objective response rates. Additional measures include duration of response, progression-free survival, and overall survival, with monitoring lasting up to approximately 63 months.

Researchers are evaluating new treatments for advanced ovarian cancer in women who have completed initial surgery and chemotherapy. The study focuses on non-HRD positive ovarian cancer, comparing a targeted therapy called sacituzumab tirumotecan sac-TMT given alone or with bevacizumab, against standard care options such as bevacizumab maintenance or observation. The goal is to see if sac-TMT with or without bevacizumab can help patients live longer without their cancer worsening. Participants in the experimental group will receive sac-TMT through intravenous infusion on days 1, 15, and 29 of every 6-week cycle until the cancer progresses, side effects become prohibitive, or other reasons for stopping arise. They may optionally receive bevacizumab on days 1 and 22 of each cycle. The comparator group will either receive bevacizumab alone every 3 weeks for up to 22 courses or be monitored without active treatment. Supportive medications like steroid mouthwash and other rescue drugs are recommended before sac-TMT infusions. Throughout the study, participants will be regularly monitored for how long they live without their disease progressing, overall survival, side effects, and quality of life using specialized questionnaires. These assessments will continue for up to approximately 78 months. The study is randomized, with single masking, and led by Merck Sharp & Dohme LLC. Participants can expect regular visits for treatment and monitoring during this period.

Researchers are evaluating AZD8421 alone and in combination with targeted anti-cancer drugs in patients with ER HER2- advanced breast cancer and metastatic high-grade serous ovarian cancer. This first-in-human study aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of AZD8421, both as a single treatment and combined with other therapies, in participants previously treated for these cancers. The study includes two main treatment modules AZD8421 monotherapy to determine a recommended dose in patients with specific advanced breast and ovarian cancers, and AZD8421 combined with camizestrant and CDK46 inhibitors in advanced breast cancer patients. Participants receive these treatments in cycles, with safety and response monitored closely. The study uses a sequential model to evaluate these approaches. Participants will undergo various assessments including clinical lab tests, vital signs, ECGs, and tumor measurements to monitor treatment effects and safety throughout the study, which includes an approximately 18-month safety follow-up. Researchers will track dose-limiting toxicities, adverse events, response rates, tumor changes, and pharmacokinetic profiles during treatment and follow-up periods.

Researchers are evaluating the long-term safety and tolerability of JNJ-81201887, given as an intravitreal injection a shot into the eye, in participants with Geographic Atrophy GA secondary to Age-related Macular Degeneration AMD. This study is a long-term extension of parent clinical trials where participants had previously received either low or high doses of JNJ-81201887 or a sham procedure. The goal is to monitor participants over an extended period to understand any lasting treatment effects or side effects. Participants entering this long-term extension study will not receive additional doses of the study drug or any new intervention as part of this trial. They previously participated in parent studies where they were treated with either low dose or high dose JNJ-81201887 or sham procedure. Some participants who were in the sham group of the parent study may receive open-label treatment outside this study before entering this extension. This study focuses solely on follow-up without new treatment administration. Throughout the study, participants will undergo regular assessments to monitor ocular and systemic safety. These include tracking treatment-emergent adverse events, clinical laboratory tests, retinal imaging, and eye examinations over up to five years. This extended monitoring aims to evaluate the long-term safety profile of the previous treatments. Participants will be followed with periodic visits and evaluations, with the total study duration extending until 2030.

Researchers are studying Friedreichs Ataxia FA, a genetic condition, to assess the safety, tolerability, and how the body processes a drug called DT-216P2. This phase 12 open-label trial focuses on understanding these factors when the drug is given in multiple increasing doses to people with FA. The study is sponsored by Design Therapeutics, Inc. and aims to gather important information about the treatments effects in this patient group. Participants will receive DT-216P2 through subcutaneous under the skin or intravenous into a vein administration in varying doses. The study includes multiple dose levels to observe the drugs behavior and side effects up to 12 weeks. There is no placebo group, and all participants will be treated with DT-216P2 during the study period, which starts in June 2025 and is planned to conclude by March 2027. During the study, participants will be closely monitored for any side effects or adverse events, with particular attention to treatment-emergent adverse events up to week 12. Researchers will also measure drug levels in the body, including maximum concentration and how long it stays in the system. Frataxin protein levels, important in FA, will be checked before and after treatment. Participants will be regularly assessed for safety and treatment effects throughout the study duration.

Researchers are evaluating the safety, tolerability, and dosing of three drugsAZD2284, AZD2287, and AZD2275in patients with metastatic castration-resistant prostate cancer. This first-in-human, Phase I trial aims to understand how these drugs behave in the body and to explore their potential effects in this advanced prostate cancer population. The study is divided into two parts Part A focuses on imaging and dosing optimization, while Part B focuses on dose escalation and therapeutic evaluation. Participants in Part A receive AZD2287 alone or combined with AZD2275 to find the best dosing approach. In Part B, different doses of AZD2284 are given based on findings from Part A, with several dose levels tested to assess safety and potential effectiveness. Expansion cohorts in Part B further explore the effects of AZD2284 at selected doses. Throughout the study, participants will undergo imaging scans such as SPECTCT, laboratory tests, and monitoring for side effects and drug levels. Researchers will track adverse events, dose-limiting toxicities, and radiation doses, as well as tumor uptake of the drugs. Longer-term outcomes like prostate-specific antigen response, progression-free survival, and overall survival will be followed for up to five years. The total duration of follow-up varies by study part and dose level.

Researchers are evaluating the safety, tolerability, and effectiveness of up to three doses of KIO-301 given by injection into both eyes every six weeks in adults with late-stage retinitis pigmentosa RP. This study focuses on patients with very limited or no light perception vision, including those classified as No Light Perception or Low Vision. Participants will be randomly assigned to receive either 50 micrograms or 100 micrograms of KIO-301, or a placebo, all administered by intravitreal injection in both eyes three times every six weeks. The main study includes a 12-week treatment period followed by a 12-week follow-up for monitoring. Those who received placebo may opt to join an additional 24-week open-label extension phase. Throughout the study, participants will attend visits every three weeks for safety, tolerability, pharmacokinetic, and efficacy assessments. The screening period can last up to 45 days, and total participation in the main study may last up to 30 weeks, with additional time if joining the extension. Researchers will closely monitor vision changes and side effects to assess the treatments impact and safety.

Researchers are evaluating the safety, tolerability, and effectiveness of a one-time intravitreal injection of SAR446597 in people with Geographic Atrophy GA caused by Age-related Macular Degeneration AMD. This sequential Phase 12 study includes two parts and will take place at multiple centers. The study aims to learn how well this treatment works and how safe it is for participants with this eye condition. Participants will receive a single intravitreal injection of SAR446597, with different dose levels tested across successive groups in Part I. In Part II, participants may receive SAR446597 at one of two dose levels or a sham injection as a control. The core study phase lasts about 2 years per participant, followed by an additional 3-year Extended Follow-Up phase to monitor long-term effects. Throughout the study, participants will undergo regular assessments including eye exams to measure changes in the size of GA lesions and visual acuity using standardized charts. Safety will be monitored closely by tracking any treatment-related adverse events. The total participation time spans approximately 5 years, allowing researchers to evaluate both short- and long-term outcomes of SAR446597 treatment.

Researchers are evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of mosunetuzumab Lunsumio alone or combined with venetoclax in adults with relapsed or refractory chronic lymphocytic leukemia CLL or small lymphocytic lymphoma SLL. The study includes participants who have had prior treatments including BTKi andor venetoclax, and some participants progressing on BTKi may continue that therapy during initial mosunetuzumab treatment. This is a phase 1B, open-label study involving multiple treatment groups, including an additional arm for non-US participants receiving combination therapy or standard-of-care regimens. Participants will receive mosunetuzumab by subcutaneous injection, either alone or combined with venetoclax or with ongoing BTKi therapy for the first three cycles. The study includes dose escalation and dose expansion phases, with one arm also receiving the standard rituximab plus venetoclax regimen for comparison. Tocilizumab may be administered intravenously as needed to manage cytokine release syndrome. Treatments are tailored based on prior therapies and disease progression, with some arms limited to non-US participants. Throughout the study, participants will be closely monitored with regular assessments for safety and treatment response, including measuring dose-limiting toxicities, objective response rates, progression-free survival, overall survival, and adverse events. Pharmacokinetic parameters of mosunetuzumab, BTKi, and venetoclax will be evaluated. Participants may undergo laboratory tests, imaging, and clinical evaluations over periods of up to 12 months or 24 months depending on the study arm. The total duration includes screening and treatment phases with ongoing safety follow-up.

Researchers are evaluating the experimental drugs pozelimab and cemdisiran for treating Geographic Atrophy GA, a late stage of Age-related Macular Degeneration AMD that affects central vision. The study aims to compare the progression rate of GA in patients receiving cemdisiran alone, the combination of pozelimab and cemdisiran, or a placebo. Additional goals include monitoring side effects, drug levels in the blood over time, and the bodys antibody response to these drugs. Participants will receive subcutaneous injections of either pozelimab combined with cemdisiran, cemdisiran alone, or a placebo. The study is randomized and double-masked with three groups receiving different treatments. Treatment and monitoring will continue through specified time points up to 104 weeks, with follow-up on safety and antibody responses extending even further. During the study, participants will attend regular clinic visits for eye exams, imaging using Fundus Autofluorescence to measure GA lesion growth, vision tests including visual acuity and contrast sensitivity, and blood tests to assess drug levels and antibody formation. Researchers will track treatment-emergent adverse events and evaluate changes in vision and GA progression over time. Participation lasts until the study completion date in April 2033, with primary outcomes assessed at 52 weeks and further evaluations up to 296 weeks.