Syracuse

Researchers are conducting an open-label, multi-center, non-randomized pivotal Phase 3 study to evaluate the effectiveness and safety of PET imaging using [18F]PI-2620 for detecting tau protein buildup in people with Alzheimer's disease and control subjects. The study compares PET imaging results during life with brain tissue analysis obtained after death through autopsy, aiming to improve diagnosis of tau-related brain changes. Participants will receive an intravenous injection of the radioligand [18F]PI-2620 at a dose of 185 MBq 20%. The PET imaging will be performed to visualize tau deposits in the brain. This study focuses on assessing the diagnostic accuracy of this imaging method by comparing it to post-mortem histopathology findings. Throughout the study, participants will undergo PET scans and assessments to determine the presence and extent of tau pathology. The primary outcome measure is the ability of visual assessment of [18F]PI-2620 PET images to correctly distinguish tau neurofibrillary pathology associated with Alzheimer's disease, confirmed at autopsy within about one year. Safety and tolerability during imaging procedures will also be monitored, with a total participation period depending on the timing of brain autopsy after death.

Researchers are looking for ways to treat germinal center B-cell-like diffuse large B-cell lymphoma (GCB DLBCL). DLBCL is a fast-growing blood cancer that affects B-cells. GCB is a type of DLBCL that affects young B-cells that are still maturing. The goal of this study is to learn if more people who receive zilovertamab vedotin (MK-2140) and R-CHP have the cancer respond (go away) than those who receive polatuzumab vedotin and R-CHP.

Researchers are investigating whether buntanetap/Posiphen can help treat early Alzheimer's disease in adults aged 55 to 85 years. This Phase 3 study aims to find out if buntanetap/Posiphen improves thinking abilities and daily functioning compared to a placebo. It also evaluates the safety of buntanetap/Posiphen by monitoring any medical issues that participants may experience during the trial. Participants will take either a 30 mg capsule of buntanetap/Posiphen or a placebo capsule by mouth once daily for 18 months. The study includes regular clinic visits at screening, enrollment, and months 1, 3, 6, 9, 12, 15, and 18. During some visits, participants will have brain MRI scans. The study uses a double-blind design, meaning neither participants nor researchers know who receives the active drug or placebo. Throughout the study, participants will complete tests and questionnaires to measure cognitive function and daily living activities, including the ADAS-Cog13 and ADCS-iADL scales. Phone calls before and after visits help track progress and adherence. Safety is closely monitored with ongoing assessments from screening through the 18-month treatment period.

Researchers are evaluating the safety and tolerability of NKX019, an investigational allogeneic CD19-directed CAR natural killer (CAR NK) cell therapy, in adults with active lupus nephritis or primary membranous nephropathy. This Phase 1/2 open-label, multi-center, non-randomized study also explores preliminary efficacy, pharmacokinetics, and pharmacodynamics of NKX019 in people with these autoimmune diseases. Participants have conditions that have persisted despite standard treatments and meet specific disease activity and biopsy criteria. Participants undergo a dose escalation phase using a 3+3 design to identify recommended doses. Each treatment cycle includes lymphodepletion with fludarabine and cyclophosphamide (Flu/Cy), though some may receive cyclophosphamide alone if cytopenic, followed by three doses of NKX019. The study monitors safety during and after treatment, and additional participants may be enrolled across indications once doses are established. During the study, participants receive medical evaluations including safety assessments for treatment-emergent adverse events and dose-limiting toxicities. Researchers assess disease activity, laboratory markers, and immunologic responses. Monitoring continues from the first NKX019 dose until 30 days after the last treatment. The study spans adults aged 18 to 70 years with specific autoimmune kidney diseases meeting detailed inclusion and exclusion criteria to ensure safety and appropriate participation.

Researchers are evaluating the safety and tolerability of NKX019, an investigational allogeneic CD19-directed CAR natural killer (CAR NK) cell therapy, in participants with autoimmune diseases such as systemic sclerosis, idiopathic inflammatory myopathies, and antineutrophil cytoplasmic antibody-associated vasculitis. This Phase 1/2, open-label, multi-center, non-randomized study uses a dose escalation and dose expansion design to assess preliminary efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of NKX019 in these immune-mediated conditions. The study uses a "3+3" dose escalation design to determine recommended doses for further study. Participants receive a cycle that starts with lymphodepletion using fludarabine and cyclophosphamide (Flu/Cy), or cyclophosphamide alone if they are cytopenic, followed by three doses of NKX019. The trial includes multiple cohorts to enroll additional participants across different autoimmune disease indications and monitors treatment effects and safety throughout. Participants undergo screening and receive treatments under close observation. Researchers monitor safety outcomes including dose-limiting toxicities during the first 28 days after the initial NKX019 dose and treatment-emergent adverse events from the first dose until 30 days after the last treatment. The study collects data on clinical responses, laboratory tests, and immune effects throughout the treatment and follow-up periods, with participant involvement spanning screening, treatment, and safety monitoring phases.

Researchers are evaluating the safety and preliminary effectiveness of a single treatment using inhibitory nerve cells called NRTX-1001 for adults with drug-resistant bilateral mesial temporal lobe epilepsy (MTLE). This open-label, multicenter Phase 1/2 study aims to reduce seizure frequency by delivering these cells directly into both temporal lobes of the brain. NRTX-1001 secretes the inhibitory neurotransmitter GABA to help suppress seizure activity, with safety and symptom effects monitored over time. Participants will receive a one-time, stereotactic CT or MRI-guided injection of NRTX-1001 cells into both temporal lobes. NRTX-1001 is made from human stem cells transformed into inhibitory interneurons designed to last long-term without needing repeat treatments. The study includes a two-year period of regular assessments approximately every three months following treatment, with continued yearly follow-up visits and phone calls from years 3 through 15 to monitor ongoing safety and seizure control. During the study, participants will undergo evaluations including seizure monitoring and safety assessments to track any serious adverse events up to 12 months after treatment. Researchers will also assess tolerability and effects on epilepsy symptoms throughout the study and long-term follow-up period. The total participation time may extend up to 15 years with regular contact to ensure safety and collect data on seizure frequency and treatment impact.

Researchers are evaluating the effectiveness of pemigatinib in adults with advanced or metastatic pancreatic cancer that has spread to nearby tissues, lymph nodes, or distant body parts, and that have specific genetic changes in the FGFR gene. The study focuses on patients whose cancer has FGFR2 gene fusions or other FGFR alterations, aiming to see if pemigatinib can block these abnormal gene functions to stop tumor growth and possibly improve quality of life. This is a phase II trial conducted nationwide using a fully decentralized telemedicine approach to reach participants. Participants receive pemigatinib as an oral medication once daily for 14 days within each 21-day cycle. Treatment continues unless the disease progresses or unacceptable side effects occur. Alongside the drug treatment, patients undergo various imaging tests including CT scans, MRI, optical coherence tomography (OCT), and when needed, whole body bone scans and dilated eye exams (ophthalmoscopy). After finishing treatment, patients are followed up at 30 days and then every four months for one year to monitor their condition. Throughout the study, patients provide blood samples and undergo scans to evaluate treatment response and detect resistance mutations. Researchers track the overall response rate for up to 24 months and assess safety and tolerability. Patients must comply with scheduled visits, tests, and oral medication intake. The total study participation includes treatment cycles and a follow-up period lasting up to approximately 16 months after treatment completion.

Researchers are evaluating the safety and effectiveness of Vagus Nerve Stimulation (VNS) Therapy as an additional treatment compared to no stimulation in people with treatment-resistant depression. This prospective, multi-center, randomized, controlled, blinded trial focuses on reducing depressive symptoms over 12 months using multiple depression rating scales. The study follows guidelines from the Centers for Medicare and Medicaid Services regarding evidence development for this treatment. Participants receive implantation of the VNS device, which delivers stimulation to the vagal nerve. After a minimum two-week period post-implantation, participants are randomly assigned to either active VNS treatment or no stimulation control, with outcomes observed for 12 months. Following this randomized phase, all participants enter an open-label extension where those in the control group receive active stimulation. Additional subjects may join this open-label study for up to five years to further assess long-term effects. Throughout the study, participants undergo regular assessments including the Montgomery Åsberg Depression Rating Scale (MADRS), WHO Disability Assessment Schedule, Health Outcome Scale, Clinical Global Impressions Scale, and Suicidality Tracking Scale. Researchers monitor response rates, remission times, duration of effects, and adverse events from implantation through 12 months. This comprehensive evaluation includes safety monitoring and functional outcome measures to understand the impact of VNS therapy on depression and related disabilities.

Researchers are evaluating how well two new study drugs, CagriSema and cagrilintide, help children and adolescents with excess body weight lose weight. This trial includes participants aged 8 to less than 18 years who have overweight or obesity. The study is designed in two parts: a main study and an extension study. The main study compares CagriSema, cagrilintide, semaglutide (an already approved drug), and placebo, with treatments assigned randomly. Participants receiving semaglutide will not continue to the extension study. The total time in the main study is about 1 year and 6 months, while those in the extension study may participate for up to about 4 years and 10 months. Participants in the main study will receive one of the four treatments by subcutaneous injection. In the extension study, participants will receive either CagriSema or cagrilintide. The study drugs are monitored closely for safety, and participants may experience side effects. The study compares these new treatments to a placebo and an existing approved drug to better understand their effects on weight management in young people. During the study, researchers will measure changes in body mass index (BMI) from baseline to week 68 as the primary outcome. Participants will undergo various assessments including laboratory tests and physical evaluations. The study tracks adherence to treatment and monitors safety throughout the study period. This comprehensive approach aims to provide detailed information about the efficacy and safety of these medications for managing weight in children and adolescents.

Researchers are evaluating oral letermovir in newborns with symptomatic congenital Cytomegalovirus (CMV) infection in a Phase 1 open-label study. The study aims to understand how letermovir behaves in the body (pharmacokinetics) and to assess its safety in infants with CMV. This condition involves symptoms such as low platelets, liver and spleen enlargement, growth problems, nervous system issues, hearing loss, and positive CMV tests in bodily fluids. The study enrolls two groups of infants. Group 1 will receive a single dose of oral letermovir followed by detailed blood testing over 24 hours to measure drug levels. Depending on these results, infants may start a 14-day daily course of letermovir at the same or adjusted doses. Oral valganciclovir, a standard treatment, will begin within the first month of life either before or alongside letermovir. Group 2 infants will start the 14-day letermovir course and valganciclovir simultaneously, with doses adjusted based on Group 1 results. Blood samples for safety labs and viral load testing will be collected multiple times during and after treatment. Participants will have blood tests on Days 1, 5, 10, and 14 of treatment, with additional viral load monitoring on Days 21 and 42 to watch for virus return after stopping letermovir. Full drug level profiles for letermovir and ganciclovir will be checked on Day 10, with additional limited sampling on other days. Adverse events will be reviewed throughout treatment and follow-up. After completing the study period, infants will continue valganciclovir for an expected total therapy duration of six months. The main measure is the amount of letermovir in the blood over 24 hours after dosing.