North Melbourne

Researchers are evaluating the gene therapy FLT201 in adults with Gaucher disease Type 1 who have been on stable enzyme replacement therapy ERT or substrate reduction therapy SRT for at least two years. This Phase 3, non-randomized, multicenter study aims to confirm the efficacy and safety of FLT201 after participants stop their current treatments. FLT201 is an advanced therapy designed to address this rare genetic condition by delivering a gene using a recombinant adeno-associated virus vector. Participants receive a single intravenous infusion of FLT201 during the study. This gene therapy uses a replication-incompetent viral vector to introduce the therapeutic gene. The study includes only one treatment group and does not involve placebo or comparison arms. The primary treatment period focuses on evaluating FLT201s impact over one year after stopping prior therapies. Throughout the study, participants undergo regular assessments to monitor their health and treatment effects. Researchers evaluate the therapys efficacy by measuring clinical outcomes at one year. Safety is closely observed to identify any adverse effects. The total study duration extends to 2032, allowing long-term follow-up of participants responses and overall well-being.

Researchers are evaluating the safety, effectiveness, and dosimetry of two treatments, 177LuLu-A9-0631 and 225AcAc-A9-0642, in adults with locally advanced, unresectable, or metastatic solid tumors that express the Gastrin-Releasing Peptide Receptor GRPR. This multicenter Phase 1-1b study focuses on several cancers including breast, prostate, colorectal, and other GRPR-positive tumors. The study aims to find safe doses and assess how these treatments affect tumors and normal organs. The study consists of two parts Phase 1 involves increasing doses to determine safety, tolerability, and the recommended dose for Phase 2. Phase 1b expands to more participants to further evaluate safety, efficacy, and dosimetry at the recommended dose. Treatments are given intravenously, and diagnostic imaging with 68GaGa-A9-6217 or low-dose 177LuLu-A9-0631 helps confirm GRPR expression and monitor responses. Participants will undergo multiple assessments including imaging scans, laboratory tests, and safety evaluations throughout the study. The main outcomes measured are the frequency and severity of side effects, dose-limiting toxicities, and response rates. Dosimetry will be assessed up to 7 days after each injection over several treatment cycles, and participants will be monitored for up to 12 months after treatment to track disease progression and treatment effects.

Researchers are evaluating nipocalimab, a drug given intravenously or subcutaneously, for adults with generalized myasthenia gravis gMG, a condition causing muscle weakness. This Phase 3 study aims to compare the effectiveness and safety of nipocalimab against a placebo and to understand how the drug works in the body when given by injection under the skin versus through a vein. The study includes a subcutaneous substudy to explore these differences in delivery methods. Participants receive nipocalimab intravenous infusions every two weeks for up to 24 weeks during a double-blind placebo-controlled phase. Those completing this phase can enter an open-label extension, choosing to continue receiving intravenous nipocalimab or switch to the subcutaneous form given weekly for eight weeks, with the option to continue longer in a long-term extension. Placebo is also given intravenously every two weeks in the control group during the double-blind phase. Throughout the study, participants undergo regular assessments including muscle function scores, quality of life evaluations, and blood tests measuring antibody levels and drug concentration. Researchers monitor safety by tracking adverse events and changes in clinical and laboratory parameters. The study measures changes in activities of daily living related to myasthenia gravis over time. Participation may last several years, as long-term monitoring continues after the initial treatment phases to evaluate ongoing effects and safety.

Researchers are studying the Akyva System, an implantable device designed to electrically stimulate the bladder muscle to help people with underactive bladder symptoms. The study aims to evaluate the safety of this device and observe any possible side effects. This research includes adults with non-neurogenic or spinal cord injury-related underactive bladder and investigates if the device can improve bladder emptying and reduce leftover urine after urination. Participants will receive the Akyva System implanted through surgery. The device targets the bladder muscle that controls urination by electrical stimulation. The study includes both non-neurogenic and neurogenic underactive bladder patients. The surgical feasibility of implanting the device will also be assessed. This is a single-group interventional trial evaluating the devices effect and safety over time. During the study, participants will be monitored from enrollment through 18 months. Researchers will assess safety related to device implantation and use, bladder emptying effectiveness, and surgical usability. Participants will be followed with bladder diaries, clinical evaluations, and safety assessments. The study involves pre-operative evaluation, device implantation, and multiple follow-up visits to measure outcomes and monitor for any adverse events.

Researchers are evaluating a new imaging agent called MNPR-101-DFO*-89Zr in patients with various solid tumor cancers, including bladder, triple-negative breast, lung, colorectal, gastric, ovarian, and pancreatic cancers. This open-label pilot study aims to assess how well this agent detects tumors using PETCT scans, its safety, and how it behaves in the body. The study is sponsored by Monopar Therapeutics and focuses on cancers that express the urokinase plasminogen activator receptor uPAR. Participants will receive a single intravenous injection of MNPR-101-DFO*-89Zr on Day 1 with a dose adjusted between 37 to 74 MBq and antibody mass dose increased stepwise to a maximum of 80 mg. After injection, three PETCT scans are performed about 2 hours post-infusion on Day 1, once between Days 3 to 5, and once between Days 7 to 10. Blood samples will be collected six times at specific intervals post-infusion to analyze pharmacokinetics. Dosimetry and tumor uptake values will be measured and compared to prior 18F-FDG PET scans. Participants will be monitored for adverse effects up to 30 days after infusion. The study includes safety assessments, measurement of tracer distribution in organs and tumors, and blood sample analysis. Each PET scan takes about 30 minutes, and the total involvement lasts approximately one month following the single infusion. Researchers will evaluate dosimetry, biodistribution, tumor uptake, and safety to understand the imaging agents potential for future clinical use.

Researchers are evaluating the safety and dosing of MNPR-101-PCTA-177Lu, a radioactive drug, in adults with solid tumor cancers who previously participated in a related imaging study. This Phase 1a, open-label, multi-center study focuses on increasing doses gradually to find safe levels while monitoring side effects and treatment impact. The study uses a special method called TITE-BOIN to decide dose changes based on patient responses. Participants will receive three intravenous infusions of MNPR-101-PCTA-177Lu over 20 minutes each, given on Cycle 1 Day 1, Cycle 1 Day 15, and Cycle 2 Day 1, with doses ranging from 480 to 2240 MBq depending on dose level. The treatment cycles last 12 weeks, and dosing decisions for patient groups of two are made using dose escalation or de-escalation rules. Imaging and scans such as SPECT and CT are performed at specific time points to assess the drugs effect on tumors. During the study, patients will have blood tests and imaging to monitor safety, side effects, and tumor response, with a follow-up period of 12 weeks after the last dose. Researchers will track dose-limiting toxicities, adverse events, and radiologic response rates using established criteria. The total study duration covers initial dosing, treatment cycles, and safety monitoring to evaluate the drugs safety profile and appropriate dosing levels.

Researchers are evaluating the effects of YB-101 on thyroid function in adults with Graves Disease. The study aims to assess how safe and well tolerated YB-101 is, as well as how it is distributed in the body. This Phase 2 trial includes participants with a documented diagnosis of Graves Disease confirmed by thyroid-stimulating hormone receptor antibodies. The study has two parts Part 1 is a blinded treatment period and Part 2 is a double-blinded treatment period. Participants will receive either YB-101 or a placebo through subcutaneous injections over a 24-week period. Those who participate in Part 1 are not eligible to participate in Part 2. The treatments and placebo are administered similarly in both parts. Participants will attend between 34 to 39 in-clinic visits over approximately 40 weeks depending on their assigned part. Researchers will monitor side effects, safety, and thyroid hormone levels including free triiodothyronine FT3, free thyroxine FT4, and thyroid-stimulating hormone TSH. They will also evaluate the proportion of participants who normalize these hormones and stop anti-thyroid drugs. The study includes safety assessments up to Day 169 and ongoing monitoring throughout the treatment period.

Researchers are evaluating the safety and effectiveness of ficerafusp alfa combined with pembrolizumab compared to placebo with pembrolizumab for adults with first-line PD-L1-positive, recurrent or metastatic Head and Neck Squamous Cell Carcinoma. This study focuses on the dual targeting of EGFR and TGF-beta, which contribute to tumor growth and spread. It includes both phase 2 and phase 3 parts to identify the best dose and to compare treatment outcomes. In phase 2, participants are randomized into three groups receiving either higher or lower doses of ficerafusp alfa plus pembrolizumab, or placebo plus pembrolizumab. In phase 3, participants receive the selected optimal biologic dose of ficerafusp alfa with pembrolizumab or placebo with pembrolizumab in a 21 ratio. Treatments are given weekly or every three weeks depending on the drug, with careful monitoring throughout the study. Participants will undergo tumor biopsies or provide archival tissue, and receive regular assessments including imaging scans and lab tests to measure tumor response and safety. Researchers will track side effects, response rates, survival, and quality of life using standardized criteria over approximately 1 to 3 years. Safety monitoring continues up to 90 days after treatment ends. The total study duration extends through long-term follow-up to evaluate overall outcomes.

Researchers are evaluating the combination of brenetafusp IMC-F106C plus nivolumab compared to standard nivolumab regimens in people with previously untreated advanced melanoma who are HLA-A*0201-positive. This phase 3, randomized, controlled study aims to understand how these treatments perform in this specific group. The study focuses on improving progression-free survival and overall outcomes for participants with advanced melanoma. Participants are assigned to one of three groups one group receives a low dose of brenetafusp once weekly for 13 weeks, then every two weeks until Week 51, and every four weeks thereafter, along with nivolumab every four weeks another group receives a high dose of brenetafusp on the same schedule plus nivolumab the third group receives nivolumab alone or nivolumab combined with relatlimab every four weeks. The study includes a dose recommendation phase with changes made in November 2025 based on safety and efficacy data. During the study, participants undergo assessments including tumor measurements per RECIST 1.1, BRAF V600 mutation status evaluation, and performance status scoring. Researchers will monitor progression-free survival up to approximately 45 months, overall survival up to 57 months, and record adverse events, immune responses, and quality of life. The study involves regular dosing and long-term follow-up to evaluate treatment effects and safety.

Researchers are evaluating the safety and effectiveness of 177Lu-TLX250, a targeted radioligand therapy, in adults with advanced, relapsed, or recurrent clear cell renal cell carcinoma ccRCC that expresses the CAIX protein. This Phase 3, randomized, open-label study aims to find the best dosing regimen and compare 177Lu-TLX250 to standard single-agent therapies used in Australia. The study includes participants who have previously received multiple systemic treatments and have measurable disease progression. The study has two parts Part 1 focuses on dose optimization by randomly assigning participants to receive either three infusions of 177Lu-TLX250 at 8-week intervals or six infusions at 4-week intervals. Part 2 compares the chosen dose of 177Lu-TLX250 with the investigators choice of a single standard therapy. Treatments are given intravenously, and the study evaluates different dosing schedules over several months. Participants will undergo various assessments including imaging scans, laboratory tests, and quality of life questionnaires throughout the study, which lasts about two years. Researchers will monitor safety, treatment response, survival, and how the drug distributes in the body. They will also evaluate side effects, immune responses, and radiation exposure. This comprehensive monitoring helps understand the therapys impact and tolerability over time.