Liverpool

Researchers are evaluating the effects of oral neflamapimod, a specific inhibitor of the enzyme p38 alpha kinase, on recovery after moderate to severe acute ischemic stroke. The study aims to determine whether neflamapimod can improve residual physical disability and cognitive dysfunction following such strokes. This is a Phase 2, double-blind, placebo-controlled clinical trial targeting adults who have recently experienced an ischemic stroke in the brain's anterior circulation. Participants will receive either neflamapimod capsules containing 40 mg of the active drug or placebo capsules that look identical but contain no active ingredients. The treatment will be administered over a 12-week period. The study compares motor recovery and other functional outcomes between the neflamapimod and placebo groups to assess the investigational drug's impact. During the study, participants will undergo various assessments including the Fugl-Meyer Assessment of Motor Recovery, the Timed Up and Go Test, and the National Institutes of Health Stroke Scale motor score. These evaluations will measure changes from baseline to Week 12 to track motor and cognitive recovery. Safety monitoring and adherence will be conducted through regular evaluations. The total participation period covers enrollment through the end of treatment at 12 weeks.

Researchers are evaluating the safety, tolerability, how the body processes the drug, and early antitumor effects of BG-C137, an antibody-drug conjugate targeting FGFR2b, alone and combined with other anticancer drugs in people with advanced solid tumors. This study includes two phases: Phase 1a focuses on dose escalation and safety, while Phase 1b involves dose expansion. The trial is sponsored by BeOne Medicines, formerly BeiGene. Participants receive BG-C137 through intravenous infusion. In combination groups, anticancer agents are given either intravenously or orally. Phase 1a includes monotherapy dose escalation, safety expansion, and combination dose confirmation and safety expansion. Phase 1b focuses on dose expansion. The study will determine the maximum tolerated dose, recommended doses for expansion, and overall response rates over approximately two years. During the study, participants will undergo evaluations including safety monitoring for adverse events, pharmacokinetic and pharmacodynamic assessments, and tumor response measurements using RECIST v1.1 criteria. Researchers will collect tumor tissue samples to assess FGFR2b expression and other biomarkers. Participants' physical function, organ health, and prior treatments will be reviewed. The total study duration may last up to about two years, with close monitoring of side effects and treatment effects throughout.

Primary immune thrombocytopenia (ITP) is a condition in which the immune system mistakenly destroys platelets, the cells that help stop bleeding. This leads to a low platelet count, making it easier to bruise or bleed. The trial investigates the long-term safety, tolerability, and effectiveness of mezagitamab in adults with chronic primary ITP who have previously participated in certain mezagitamab studies. It also examines how the body processes mezagitamab over time. Participants who completed the previous mezagitamab studies TAK-079-3002 or TAK-079-1004 and meet specific criteria will receive mezagitamab as a subcutaneous injection during this continuation study. The study is open-label and multicenter, focusing on continued treatment based on protocol requirements. The medication is given under medical supervision, and participants return to the study clinic several times throughout the study. During their participation, individuals will undergo regular assessments including monitoring for treatment-emergent adverse events and serious adverse events up to approximately 108 weeks. Researchers will track safety by noting any adverse events that lead to permanent withdrawal from mezagitamab. The study includes physical evaluations, laboratory tests, and ongoing safety monitoring to understand how well participants tolerate the treatment and how effective it is over the long term.

Researchers are conducting a phase III randomized, open-label, multicenter trial across several countries including Sweden, Norway, Finland, Denmark, Italy, Australia, and New Zealand. The study focuses on elderly patients with untreated diffuse large B-cell lymphoma (DLBCL), defined as patients aged 80 years or older, or those aged 75 years or older who are considered frail based on a simplified Comprehensive Geriatric Assessment. The trial aims to compare the effectiveness of two treatment regimens in this population. Participants are randomly assigned to receive either the standard R-miniCHOP treatment or an experimental R-pola-miniCHP regimen where vincristine is replaced with an immunoconjugate, polatuzumab vedotin. Both treatments involve cycles of drugs including rituximab, cyclophosphamide, doxorubicin, and prednisone, administered over 18 weeks. The trial includes a screening period lasting up to 4 weeks, followed by the active treatment phase, and then a follow-up period lasting up to 36 months after treatment completion. Throughout the study, participants will be monitored to measure progression-free survival over 2 years as the primary outcome. The study involves regular assessments including clinical evaluations and safety monitoring. Enrollment began in the first quarter of 2020, with the last patient visit expected by the first quarter of 2027, allowing for long-term observation of treatment effects and patient outcomes.

Researchers are studying the real-world effectiveness of pegcetacoplan in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This long-term, multicenter observational study aims to fill knowledge gaps about how pegcetacoplan works in routine medical practice. It also seeks to provide important information on red blood cell transfusions and healthcare resource use before and after starting pegcetacoplan treatment. Patients who have started pegcetacoplan treatment within the last 12 months or are prescribed it at enrollment will be followed for approximately 36 months. The study will collect both retrospective data from up to 12 months before treatment start and prospective data during treatment, with a total data collection period of up to about 48 months. After stopping pegcetacoplan, patients will remain in the study for 8 weeks to monitor for any adverse events. Participants will attend their usual medical visits, and data from these visits will be collected, including effectiveness measures, safety reports, patient- and clinician-reported outcomes, and healthcare use. The primary outcome includes changes in hemoglobin levels over 6 months from the start of pegcetacoplan treatment. The study plans to enroll about 200 patients across multiple countries, and data collection includes both retrospective and prospective periods, capturing comprehensive information on pegcetacoplan use in real-world settings.

Researchers are evaluating ziltivekimab as a treatment for people living with heart failure and inflammation. This Phase 3 study compares ziltivekimab to a placebo in participants with heart failure who have mild to preserved ejection fraction and systemic inflammation. The study aims to assess the effect of ziltivekimab on cardiovascular death, heart failure hospitalization, or urgent heart failure visits over a period of up to 4 years. Participants will receive monthly injections of either ziltivekimab or a placebo using a pre-filled syringe or a pen-injector. The study medication is administered subcutaneously once a month for up to 4 years. The trial includes up to 20 clinic visits during which participants will be monitored and assessed. During the study, participants will use a study app on their phone to record all injections and complete questionnaires. Researchers will monitor participants for key outcomes like cardiovascular events and heart failure episodes from the time of randomization until the end of the study. Safety and health status will be regularly evaluated throughout the study period, which may last up to 48 months.

Researchers are evaluating AZD0120, a dual-targeted CAR-T therapy against BCMA and CD19, compared to standard treatments for participants with relapsed refractory multiple myeloma (RRMM). This Phase III, randomized, open-label global study compares AZD0120 with established regimens including DKd (daratumumab, carfilzomib, dexamethasone), DPd (daratumumab, pomalidomide, dexamethasone), PVd (pomalidomide, bortezomib, dexamethasone), and Kd (carfilzomib and dexamethasone). The study aims to assess the safety and effectiveness of AZD0120 in this patient population. Participants will receive either AZD0120 CAR-T cell therapy or one of the standard drug regimens. The treatments involve combinations of biologic and drug therapies targeting multiple myeloma. The study is designed to evaluate outcomes such as progression-free survival and minimal residual disease negativity rate over periods of up to three years. During the study, participants will be closely monitored with regular assessments to evaluate disease progression and treatment response. Researchers will track progression-free survival over three years and measure minimal residual disease negativity rates at nine months, among other safety and efficacy evaluations. Participants will have laboratory tests and clinical evaluations to ensure safety and to measure treatment effects throughout the study duration.

Researchers are evaluating efruxifermin (EFX) in adults aged 18 to 80 who have compensated cirrhosis caused by nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH). This Phase 3, randomized, double-blind, placebo-controlled study aims to assess the safety and effectiveness of EFX in improving liver health and delaying disease progression in this population. The study focuses on subjects with advanced liver fibrosis (stage 4) but without liver decompensation. Participants are randomly assigned to receive either efruxifermin or a placebo, both administered by subcutaneous injection. The study includes two cohorts: Cohort 1 requires biopsy confirmation of liver fibrosis and specific metabolic features, while Cohort 2 allows biopsy or non-invasive diagnosis. Treatment and observation continue over an extended period to evaluate changes in liver fibrosis and clinical events. During the study, researchers will monitor the time until significant clinical events such as disease progression or liver decompensation occur, with a follow-up of up to five years. For Cohort 1, the proportion of participants showing improvement in fibrosis without worsening steatohepatitis will be assessed at 96 weeks. Participants will undergo regular evaluations including clinical assessments and laboratory tests to track liver function and safety throughout the study period.

Researchers are investigating the safety and effectiveness of efruxifermin in people with non-cirrhotic nonalcoholic steatohepatitis (NASH) or metabolic dysfunction-associated steatohepatitis (MASH) who have moderate to advanced liver fibrosis (stage 2 or 3). This Phase 3 study is randomized, double-blind, and placebo-controlled, enrolling a total of 1650 participants in two groups to evaluate treatment outcomes. Participants will receive either efruxifermin or a placebo by subcutaneous injection. The study involves two cohorts, with Cohort 1 including patients who have biopsy-confirmed NASH or MASH and specific liver fibrosis and activity scores. The treatment period and detailed dosing schedules are not provided but the study compares the effects of the active drug against placebo. During the study, participants will be monitored for improvement in liver disease status, including resolution of NASH/MASH and at least a one-stage improvement in liver fibrosis after 52 weeks for Cohort 1. Long-term outcomes such as event-free survival will be observed over 240 weeks. Safety and efficacy assessments will be conducted throughout the study period, including evaluations of liver histology and metabolic health.

Researchers are evaluating the safety and effectiveness of divarasib combined with pembrolizumab compared to pembrolizumab with pemetrexed and either carboplatin or cisplatin. The study focuses on adults with advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) that has a specific KRAS G12C mutation. This is a Phase III trial aiming to improve first-line treatment options for these patients. Participants will receive one of two treatment combinations. One group will take divarasib orally once daily along with pembrolizumab given through an intravenous infusion every three weeks. The other group will receive pembrolizumab with pemetrexed and either carboplatin or cisplatin, all administered by intravenous infusion every three weeks. Treatment schedules and dosages are carefully monitored during the study. Throughout the study, participants will be regularly assessed for progression-free survival and overall survival, with follow-up lasting up to approximately five years. Researchers will perform various evaluations including tumor measurements and safety monitoring. This long-term observation helps to understand the treatments' effects and safety over time, supporting informed decisions for future lung cancer therapies.