Deerfield Beach

Researchers are investigating the safety and effects of 4D-150 gene therapy in adults aged 50 and older with neovascular (wet) age-related macular degeneration (AMD) who are already receiving anti-VEGF treatment and have shown a clinical response. This Phase 1/2 trial includes dose-escalation and randomized, controlled, masked expansion phases, aiming to evaluate 4D-150 administered by intravitreal injection in one eye, with additional substudies assessing dosing in the second eye and vector shedding. Participants will receive a one-time dose of 4D-150 by injection into the study eye, followed by monthly assessments for 24 months to monitor safety and effectiveness. Those who receive 4D-150 will then enter a long-term follow-up period up to 5 years to assess ongoing safety and the duration of treatment effects. Substudies include one for contralateral eye dosing and another to characterize vector shedding, with participants monitored regularly for safety through one year and continuing long-term follow-up through year 5. Throughout the study, participants will undergo tests of visual and retinal function and structure, with assessments performed monthly initially and safety monitored for up to five years. Researchers will track treatment-emergent adverse events, serious adverse events, and any significant changes in safety parameters. Participants must comply with study procedures and visits, and males receiving 4D-150 will be advised to use barrier methods during intercourse for six months to prevent fluid transmission.

Researchers are investigating the similarity in pharmacokinetic (PK) profile, effectiveness, safety, and immune response of HLX17 compared to US-sourced Keytruda® in patients who have had surgery for non-small cell lung cancer, melanoma, or renal cell carcinoma. This Phase I, multicenter, randomized, double-blind study aims to compare these two treatments in people with these resected solid tumors to better understand their performance and safety profiles. Participants will receive either HLX17 or US-sourced Keytruda®, each given at a dose of 200 mg on the first day of every 3-week cycle. The study is designed with parallel groups, where each participant receives one of the treatments across multiple cycles. The dosing schedule continues through six cycles, and the two treatments are directly compared under controlled conditions. Throughout the study, participants will be monitored closely with various assessments including laboratory tests and evaluations of organ function to ensure safety. The main outcomes measured are drug exposure over time from the first dose to 21 days after the initial and sixth doses. Participants are expected to have a performance status of 0 and a life expectancy of at least 12 weeks. Safety and immunogenicity will also be evaluated, with follow-up to monitor any side effects or immune responses during and after treatment.

Researchers are evaluating the safety and effectiveness of HLX22 combined with trastuzumab and chemotherapy as the first treatment for patients with HER2-positive locally advanced or metastatic adenocarcinoma of the gastric or gastroesophageal junction. This phase 2, double-blind, randomized, and multiregional study compares this combination against trastuzumab and chemotherapy with or without pembrolizumab. The study aims to measure how well the treatments work in controlling the disease and improving survival for up to five years. Participants will be randomly assigned to one of two groups. One group receives HLX22 at 15 mg/kg every three weeks along with trastuzumab, chemotherapy (XELOX regimen), and possibly a placebo for pembrolizumab. The other group receives a placebo for HLX22 plus trastuzumab, chemotherapy (XELOX), and possibly pembrolizumab every three weeks. Treatment continues until the disease worsens, unacceptable side effects occur, withdrawal of consent, or other protocol-specified reasons. Throughout the study, participants will undergo regular assessments including tumor scans reviewed by an independent committee to evaluate progression-free survival and overall survival over up to five years. Other evaluations include safety monitoring and organ function tests. The study tracks how long patients live without disease progression and overall survival, aiming to better understand the benefits and risks of HLX22 combined with current standard treatments.

Researchers are evaluating the effects of ASTX727 combined with iadademstat versus ASTX727 alone in patients with accelerated or blast phase Philadelphia chromosome-negative myeloproliferative neoplasms (MPNs), including subtypes like polycythemia vera and myelofibrosis. This phase II trial aims to compare the complete acute leukemia response rate within four 28-day treatment cycles and assess survival outcomes and transplant rates. The study also explores molecular changes and resistance pathways related to these treatments. Participants are randomly assigned to one of two groups: one receives ASTX727 alone, which is a combination of decitabine and cedazuridine taken orally once daily on days 1 through 5 of each cycle; the other group receives the same ASTX727 dosing plus iadademstat taken orally on days 1-5, 8-12, 15-19, and 22-26. Treatment cycles repeat every 28 days until disease progression or unacceptable side effects occur. The study includes a dose escalation phase before randomization. During the study, participants undergo buccal swab, blood sample collections, and bone marrow aspiration and biopsy to monitor disease and treatment effects. After stopping treatment for reasons other than disease progression, patients are followed up every three months; if stopping due to progression, follow-up occurs every six months. Researchers measure treatment response using established leukemia criteria and track safety and overall survival throughout the study.

Researchers are evaluating the safety and effects of a medicine called disitamab vedotin for adults with advanced breast cancer that is hard to treat and has spread in the body. This study focuses on participants whose tumors express HER2 and who have received previous treatments for their advanced breast cancer. The goal is to understand how well this medicine works and its safety in these patients through a Phase 1b/2 open-label study. All participants will receive disitamab vedotin intravenously (IV) once every two weeks at the study clinic. They will continue the treatment until they or their doctor decide to stop, which could be due to cancer progression, side effects, or personal choice. During treatment, study visits occur every two weeks. After stopping treatment, participants will have follow-up visits about every six weeks, and later follow-up phone calls approximately every twelve weeks. Participants will undergo evaluations including assessments of their cancer response by the study doctors, following recognized criteria. The study team will monitor the participants for up to about two years or until their disease progresses or they pass away. This includes safety monitoring and collecting information about the medicine’s effects to determine its safety and effectiveness.

Researchers are evaluating the safety and effectiveness of Raludotatug Deruxtecan (R-DXd) in people with platinum-resistant, high-grade ovarian, primary peritoneal, or fallopian tube cancer. This study includes two parts: Phase 2 to find the best dose based on safety and response, and Phase 3 to compare R-DXd with the investigator's choice of chemotherapy. R-DXd is an antibody-drug conjugate that targets CDH6, a protein overexpressed in tumor cells. Participants will receive R-DXd through intravenous infusions. In Phase 2 (Part A), the dose will be optimized, and biopsies will be collected before and during treatment if possible. In Phase 3 (Part B), participants will be randomly assigned to receive either R-DXd or chemotherapy chosen by their doctor, which may include paclitaxel, topotecan, or PLD, all given by IV infusion. The study monitors treatment effects up to 18 months in Phase 2 and up to 26 months in Phase 3. During the study, participants will have regular scans and assessments to measure tumor response and progression-free survival. Researchers will monitor safety and organ function through lab tests and performance status evaluations. Participants must be willing to follow the study visits and procedures, which include biopsy samples in Phase 2 and imaging assessments to evaluate treatment response. The study aims to provide detailed information about how well R-DXd works and its safety in this patient group.

Researchers are evaluating the efficacy and safety of ML-007C-MA in treating hallucinations and delusions associated with Alzheimer's Disease Psychosis (ADP) in men and women aged 55 to 90 years. This Phase 2 study is randomized, double-blind, and placebo-controlled, aiming to compare ML-007C-MA with placebo by measuring changes in the Neuropsychiatric Inventory-Clinician: Hallucinations and Delusions (NPI-C H+D) score over seven weeks. Participants receive ML-007C-MA at doses of either 105/1.5 mg twice daily or 210/3 mg twice daily, or placebo tablets, during the treatment period. The study is designed to assess the impact of these treatments on psychotic symptoms associated with Alzheimer's disease, specifically hallucinations and delusions. Throughout the study, participants are monitored for changes in the NPI-C H+D score from baseline to the end of treatment at seven weeks. Researchers also evaluate safety and monitor participants' overall condition. The total involvement includes assessments related to cognition, symptom severity, and adherence, ensuring comprehensive evaluation of the treatment's effects and safety profile.

Researchers are evaluating two treatment combinations for patients with melanoma that has spread to the brain and has a specific BRAF-V600 mutation. This phase II trial compares encorafenib, binimetinib, and nivolumab against ipilimumab and nivolumab to determine which approach better controls and shrinks brain metastases from melanoma. The study also aims to assess overall survival, response rates, treatment duration, and side effects of each regimen. Participants are randomly assigned to one of two groups. One group receives encorafenib orally once daily, binimetinib orally twice daily, and nivolumab intravenously every 28 days. The other group receives nivolumab intravenously and ipilimumab intravenously during the first four cycles, with cycles every 21 days initially, then every 28 days thereafter. Treatment continues unless the disease worsens or side effects become unacceptable. After treatment ends, participants have follow-up visits every six months for two years, then yearly until three years after starting the study. During the trial, participants undergo brain MRIs to monitor tumor response using standardized criteria. Imaging, tumor tissue, spinal fluid, stool, and blood samples are collected for research. Safety and effectiveness are carefully assessed through scans, physical exams, lab tests, and side effect monitoring. Progression-free survival up to three years after randomization is the main outcome. Participants remain in the study for about three years with periodic evaluations to track their health and disease status.

Researchers are evaluating two surgical procedures, bilateral salpingectomy and bilateral salpingo-oophorectomy, to see how well they reduce the risk of ovarian cancer in women who have BRCA1 gene mutations. The study aims to determine if removing just the fallopian tubes (bilateral salpingectomy) is almost as effective as removing both the fallopian tubes and ovaries (bilateral salpingo-oophorectomy) in lowering ovarian cancer risk. This trial also assesses symptoms related to estrogen loss, quality of life, sexual function, cancer-related distress, decision-making about surgery, and treatment side effects in these patients. Participants choose between two groups: one group undergoes bilateral salpingectomy and may have their ovaries removed later, while the other group undergoes bilateral salpingo-oophorectomy. Both groups receive pelvic or transvaginal ultrasounds or pelvic MRI scans during screening, and blood samples are collected throughout the trial. Ancillary studies include quality-of-life assessments and questionnaires. The study also collects tissue and blood samples for future research. After surgery, participants have follow-up visits at 10 to 60 days, then at 6, 12, and 24 months, and annually for up to 20 years. Researchers monitor the time until any high-grade serous carcinomas develop, specifically ovarian, primary peritoneal, or fallopian tube cancers. They also track menopausal symptoms, sexual function, quality of life, cancer distress, medical decisions about surgery, and any adverse events during this long-term follow-up.

Researchers are evaluating the safety and effectiveness of KarXT in adults aged 55 to 90 who have mild to severe Alzheimer's Disease (AD) accompanied by moderate to severe psychosis related to AD. This phase 3 study aims to better understand how KarXT compares to a placebo in treating the psychotic symptoms associated with Alzheimer's Disease. Participants must have documented AD diagnosis and a history of psychotic symptoms lasting at least two months prior to starting the study. Participants will receive either KarXT or a placebo, with specified doses given on designated days. The study is designed as a randomized, double-blind, placebo-controlled trial with parallel groups to assess the treatment's effects. Details about dosing schedules and administration are planned but not specified here. During the study, researchers will measure changes from baseline in the Neuropsychiatric Inventory-Clinician: Hallucinations and Delusions (NPI-C: H+D) score up to week 14 to evaluate the impact on psychosis symptoms. Participants will undergo brain imaging (MRI or CT) if not already done within the past five years to rule out other conditions, and safety monitoring including laboratory tests will be conducted. The total participation duration covers screening through at least 14 weeks of treatment and assessment.