Syracuse

Researchers are evaluating the use of PET imaging with the radioligand 18FPI-2620 to detect tau protein deposits in people with Alzheimers disease and healthy controls. This open-label, multi-center, non-randomized Phase 3 study aims to compare PET imaging results during life with brain tissue analysis after death to better understand tau pathology in Alzheimers. The study is sponsored by Lantheus Biosciences Ltd. and focuses on diagnostic accuracy and safety of this imaging technique. Participants receive an intravenous injection of 18FPI-2620 at a dose of 185 MBq 20%. The study involves a PET scan procedure that participants must tolerate, including lying still in the scanner. There are no randomized groups or placebo controls as this is an open-label study. The research compares the PET imaging findings with post-mortem brain autopsy results to evaluate the ability of this imaging to detect tau deposits accurately. During the study, participants undergo PET imaging and are monitored for their ability to tolerate the scan. Brain donation consent is required for post-mortem histopathological comparison. Researchers assess the diagnostic performance of the PET scan in correctly identifying tau-related pathology and Alzheimers disease changes. The primary outcome focuses on the accuracy of visual assessment of PET images compared to autopsy findings, with follow-up continuing until study completion and an average of one year after death.

Researchers are evaluating treatments for germinal center B-cell-like diffuse large B-cell lymphoma GCB DLBCL, a fast-growing blood cancer affecting immature B-cells. The study compares two treatment combinations to see if more people respond to zilovertamab vedotin MK-2140 plus R-CHP versus polatuzumab vedotin plus R-CHP. This Phase 2 trial aims to assess the effectiveness and safety of these regimens in participants with newly diagnosed GCB DLBCL. Participants receive either zilovertamab vedotin along with rituximab, cyclophosphamide, doxorubicin, and prednisone R-CHP, or polatuzumab vedotin combined with R-CHP. Treatments are given by intravenous infusion on Day 1 of each 3-week cycle for up to 6 cycles, approximately 4 months, with prednisone or prednisolone taken orally for 5 days of each cycle. For participants with high-risk DLBCL, up to 2 additional cycles of rituximab or biosimilar are given. During the study, participants are monitored for response to treatment using Lugano Response Criteria, with follow-up lasting up to about 31 months for the primary outcome. Secondary outcomes include progression-free survival, overall survival, event-free survival, duration of complete response, adverse events, and quality of life assessments. Safety and health status are regularly checked through exams, lab tests, and questionnaires over several years, with total study participation extending up to 7 years.

Researchers are evaluating the safety and tolerability of NKX019, an investigational allogeneic CD19-directed CAR NK cell therapy, in adults with autoimmune diseases such as Lupus Nephritis and Primary Membranous Nephropathy. This Phase 12, open-label, multi-center study uses a dose escalation design to find recommended doses and assess preliminary effects, pharmacokinetics, and pharmacodynamics. Participants undergo a treatment cycle starting with lymphodepletion using fludarabine and cyclophosphamide or cyclophosphamide alone if cytopenic, followed by three doses of NKX019. The study uses a 33 dose escalation to determine safe dosing and includes dose expansion cohorts. The treatment aims to evaluate the impact of NKX019 on autoimmune disease activity and kidney function. During the study, participants are closely monitored for dose-limiting toxicities, adverse events, and lab abnormalities from the first dose until follow-up. Researchers assess kidney response, disease activity scores, and drug levels in blood for up to two years after infusion. Immunogenicity and effects on background therapies are also evaluated. The total participation time varies based on follow-up assessments and treatment response.

Researchers are studying NKX019, an investigational allogeneic CD19-directed CAR NK cell therapy, to evaluate its safety and tolerability in adults with various autoimmune diseases. This Phase 12, open-label, multi-center study uses a dose escalation and expansion design to find recommended doses and assess preliminary effects including pharmacokinetics, pharmacodynamics, and immunogenicity. Participants will undergo a treatment cycle starting with lymphodepletion using fludarabine and cyclophosphamide before receiving three doses of NKX019. A modified lymphodepletion regimen using cyclophosphamide alone may be given to participants with low blood cell counts. The study includes dose escalation using a 33 design and subsequent enrollment of more participants at the recommended dose levels. During the study, participants will be closely monitored for side effects, treatment-related toxicities, and immune responses. Researchers will collect data on lung disease, systemic sclerosis, inflammatory muscle diseases, vasculitis, and rheumatoid arthritis up to two years after NKX019 infusion. Safety assessments cover the first 28 days after dosing and continue through 30 days after the last treatment. Total participation duration may extend to two years to observe long-term effects.

Researchers are evaluating the safety and early effects of a new neuronal cell therapy called NRTX-1001 for adults with drug-resistant bilateral mesial temporal lobe epilepsy MTLE. This open-label, single-arm study involves up to 10 subjects and aims to reduce seizure frequency by targeting both temporal lobes in patients whose seizures have not improved with medication. The investigational treatment uses inhibitory nerve cells called interneurons to potentially suppress seizure activity by releasing a natural brain chemical called GABA. Participants will receive a single administration of NRTX-1001 through a stereotactic CT or MRI-guided injection directly into both temporal lobes of the brain. The therapy is designed to persist long-term, so repeated doses are not planned. This phase 12 trial observes subjects closely for safety and seizure changes over two years after treatment. Following this period, participants will continue with less frequent follow-up phone calls every three months and yearly visits through year 15 to monitor long-term outcomes. During the study, participants will undergo evaluations approximately every three months for two years, including clinical assessments and seizure monitoring. Safety and tolerability will be specifically tracked, with the main outcome being the rate of serious adverse events at 12 months post-treatment. Seizure frequency changes will also be measured as a secondary outcome. After the initial two years, ongoing safety and health status will be followed through scheduled calls and visits for up to 15 years, emphasizing long-term monitoring and support.

Researchers are evaluating DB-2304 in a Phase 12a study involving healthy adults and individuals with Systemic Lupus Erythematosus SLE or Cutaneous Lupus Erythematosus CLE. The study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of DB-2304 for injection in these participants. This trial is randomized and double-blind, sponsored by DualityBio Inc., and includes adults aged 18 to 70 years. Participants receive different dose levels of DB-2304 or placebo, with some also receiving prednisone as part of their stable treatment regimen. The study includes two parts Part A with healthy adults aged 18 to 55, and Part B with adults aged 18 to 70 having SLE or CLE. Treatments are administered by injection, and participants are randomly assigned to one of several dose levels to evaluate different dosing effects. During the study, participants will undergo monitoring for treatment-emergent adverse events, serious adverse events, and various vital signs including ECG, heart rate, respiratory rate, body temperature, and weight. These measurements are tracked for up to 112 days after treatment in Part A and up to 280 days after the first treatment in Part B. The study involves regular assessments to evaluate safety and treatment response over these periods.

Researchers are evaluating the use of pemigatinib for adults with advanced or metastatic pancreatic cancer that has spread locally or to distant parts of the body. This study focuses on patients whose cancer has specific abnormal changes in the FGFR gene, which can promote cancer growth. The goal is to see if pemigatinib can block these abnormal genes to stop tumor growth and improve quality of life. Participants take pemigatinib orally once daily for 14 days in each 21-day cycle, continuing as long as the cancer does not worsen or side effects are manageable. During the study, patients undergo blood tests, CT andor MRI scans, and optical coherence tomography OCT. Additional scans like whole body bone scans and eye exams may be performed if needed. After treatment, patients are followed up 30 days later and then every 4 months for one year. Throughout the study, researchers assess tumor response using imaging and blood tests, including monitoring cell-free DNA to track response and resistance. They measure overall response rate up to 24 months and evaluate progression-free survival, disease control, overall survival, and side effects up to 12 months. Safety and tolerability are closely monitored, and patients overall health and treatment effects are regularly checked to understand the impact of pemigatinib.

Researchers are evaluating the safety and effectiveness of the Vagus Nerve Stimulation VNS Therapy System as an additional treatment for people with treatment-resistant depression. This prospective, multi-center, randomized, controlled, and blinded trial compares active VNS therapy to a no stimulation control group in reducing depressive symptoms over 12 months. The study follows guidelines aligned with Medicare and Medicaid coverage decisions for VNS in this condition. Participants receive an implant of the VNS device and are randomized at least two weeks after implantation to either have the device activated or remain without stimulation for the first 12 months. After this initial period, those in the control group can begin stimulation. Following the 12-month randomized phase, all participants enter an open-label, longitudinal study lasting about five years, including new enrollees after the initial trial phase. During the study, participants are monitored through various depression rating scales, including the Montgomery sberg Depression Rating Scale MADRS, to assess response and remission rates up to 12 months. Safety is tracked by recording adverse events from implantation through the first year. Additional assessments include disability and health outcome scales, as well as suicidality tracking. The study aims to gather long-term data on treatment effects and participant well-being.

Researchers are gathering real-world data from patients with Philadelphia chromosome-positive chronic myelogenous leukemia in the chronic phase Ph-CML-CP who are treated with tyrosine kinase inhibitors TKIs approved for first or second line therapy. This registry study aims to collect evidence on the tolerability, safety, effectiveness, and patient-reported outcomes over a prospective follow-up period of 5 years to understand long-term treatment results in routine healthcare settings. Participants will be those receiving TKI treatments including asciminib, bosutinib, dasatinib, imatinib, or nilotinib as prescribed in standard medical care, either as initial therapy or after one prior TKI therapy. The study includes patients who began these treatments on or after specified dates and those continuing asciminib after participating in an interventional trial. There is no treatment allocation instead, patients are observed based on their current prescribed TKI therapy. During the 5-year follow-up, participants treatment continuation and discontinuation due to adverse events will be monitored, along with the occurrence of adverse events, treatment switches, molecular and hematological responses, survival outcomes, and patient-reported measures of health, symptoms, and medication adherence. Data will be collected from electronic medical records and claims data with patient consent, ensuring ongoing safety and effectiveness observations in real-world care up to 5 years.

This research aims to evaluate how well two new drugs, CagriSema and cagrilintide, help children and adolescents with excess body weight lose weight. The study includes participants aged 8 to under 18 years who have overweight or obesity. It is a Phase 3 trial that compares these new drugs with semaglutide, a drug already prescribed for weight management, and a placebo to understand their effects on weight loss. Participants in the main study are randomly assigned to receive one of four treatments CagriSema, cagrilintide, semaglutide, or placebo. All treatments are given once weekly as subcutaneous injections, starting with a dose escalation phase lasting up to 16 weeks, followed by a maintenance phase for 52 weeks. Those who receive semaglutide do not join the extension study. Participants in the extension study continue treatment with either CagriSema or cagrilintide for up to 156 weeks, while placebo participants follow a specific dosing regimen before continuing in the extension. During the study, participants will be monitored for changes in body mass index BMI and weight over time, with assessments at baseline, week 68, and for some measures, week 224. Researchers will also track body composition, metabolic markers, quality of life, and safety events. The entire duration for participants can be up to nearly five years if they take part in both the main and extension studies, involving regular visits and evaluations to understand the treatments effects and safety.