Parma

Researchers are evaluating remibrutinib (LOU064) in adolescents aged 12 to under 18 years who have chronic spontaneous urticaria (CSU) that is not well controlled by H1-antihistamines. This Phase 3 trial aims to assess the effectiveness, how the drug is processed in the body, and safety of remibrutinib compared to a placebo. The study also intends to gather long-term data on how well remibrutinib works and its safety over several years after treatment ends. The trial includes three periods. First, the core period is a 24-week double-blind phase where about two-thirds of participants receive remibrutinib and one-third receive placebo, with about 10 site visits over approximately 32 weeks. Next is an optional open-label extension lasting from one to three years, where participants who completed the core period may receive remibrutinib or enter an observational treatment-free phase depending on their symptoms. Participants may cycle through treatment and observational periods up to six times. Finally, an optional long-term treatment-free follow-up can last up to three years with one site visit and up to four phone calls. During the study, participants undergo assessments including changes in urticaria activity scores (UAS7), itching severity (ISS7), and hive severity (HSS7) measured from baseline to 12 weeks. Regular visits monitor safety, symptoms, and drug effects. The study tracks these measures to understand remibrutinib's impact on CSU symptoms and overall safety profile during and after treatment, with total participation potentially lasting several years.

Researchers are creating a national registry in Italy for multiple myeloma, a type of blood cancer that makes up about 1.3% of all tumor diagnoses in men and 1.2% in women. This registry aims to track current clinical practices and describe how patients with multiple myeloma are diagnosed and treated across various hematology centers in Italy. The study also includes a patient-powered registry to encourage patient involvement and better understand treatment patterns and outcomes. The study is observational, meaning it will not involve any experimental treatments but will collect data on routine care and outcomes for patients diagnosed with active or symptomatic multiple myeloma since January 1, 2019. Both physicians and patients will contribute information to the registry, which will help monitor standard care practices nationwide. Participants will be followed to measure important outcomes such as overall survival and the time until the next treatment over a three-year period. The registry will collect data to analyze treatment approaches, patient characteristics, and survival, helping to identify changes and differences in care across Italy. Patients aged 18 years and older who can provide informed consent are eligible to participate, and there are no exclusion criteria.

Researchers are assessing the safety and effects of marstacimab as a potential treatment for hemophilia in children aged 1 to 17 years. This study includes pediatric participants with severe Hemophilia A or moderately severe to severe Hemophilia B, with or without inhibitors. Enrollment will occur in stages, starting with adolescents aged 12 to 17 years, followed by children aged 6 to 11 years, and finally those aged 1 to 5 years. Participants must have detailed historical records of their hemophilia treatment and bleeding events for at least one year prior to joining the study. All participants will receive marstacimab once weekly by subcutaneous injection, with the first dose given at the study site by staff. During the 12-month treatment period, weekly injections may be administered at home or at the study site based on preference. The study compares participant experiences during treatment with their historical records before starting marstacimab to evaluate its potential to prevent bleeding episodes common in hemophilia. Participants will be involved for about 14 months, including a 1-month screening period, 12 months of treatment, and a 1-month follow-up. They will visit the study site at least 10 times, with the option for two visits to be conducted at home if local rules allow. Additionally, six telephone calls will be scheduled every two months. Researchers will monitor bleeding rates, adverse events, thrombotic events, immune responses, injection site reactions, and hypersensitivity throughout the study period.

This is a long-term follow-up trial to the post-authorisation efficacy and safety (PAES) trial (trial 20-HMedIdeS-19). The trial will include patients who participated in the PAES trial and were transplanted with a new kidney after treatment with imlifidase (trial drug) or standard of care medication. Imlifidase is a medicine used to prevent the body from rejecting a newly transplanted kidney and is used before transplantation in adults who have antibodies against the donor kidney and are considered 'highly sensitised' based on a positive crossmatch test. The purpose of this follow-up trial is to fulfil requirements from the European Medicines Agency (EMA) to continue to evaluate efficacy (kidney function) and safety (side effects) over time, for the patients who were transplanted with a new kidney in the PAES trial. The patients will be followed for up to 5 years after transplantation in the PAES trial to collect valuable long-term data.

Researchers are studying chronic Philadelphia-negative myeloproliferative syndromes, which are blood disorders including essential thrombocythemia, polycythemia vera, and myelofibrosis. Myelofibrosis can develop on its own or from other syndromes and is marked by bone marrow fibrosis, a sign of a more severe disease stage. The goal is to find a non-invasive way to detect fibrotic progression using cells expressing the CCR2 receptor, potentially replacing invasive bone marrow biopsies. The study evaluates a new diagnostic method combining flow cytometry analysis of blood samples and PET-CT imaging with a tracer that binds to CCR2-expressing cells. This approach aims to track and quantify these cells to identify fibrotic stages early. The project includes preclinical tests in animal models and first-in-human proof of concept using PET/CT and flow cytometry. Participants will undergo blood tests and imaging scans to measure CCR2-positive cells. Researchers will assess the percentage of circulating CD34+/CCR2+ cells at enrollment and use PET/CT scans to detect fibrotic changes. This method may help with diagnosis, monitoring disease progression, and guiding treatment decisions without the need for invasive biopsies. The study includes follow-up evaluations to support these goals.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study to see if it can reduce the risk of major adverse cardiovascular events (MACE-PLUS) in adults with established atherosclerotic cardiovascular disease (ASCVD) or those at high risk for a first ASCVD event. The study compares AZD0780 to a placebo and monitors participants from randomization until the primary analysis censoring date, followed by a final study closure visit. Participants will be randomly assigned to receive either oral AZD0780 or an oral placebo once daily. The treatment period lasts until the primary analysis censoring date, after which a study closure visit will occur. The study is event-driven and designed to assess the time to the first major cardiovascular event during treatment. During the study, participants will be closely monitored with various assessments to evaluate cardiovascular outcomes and safety over approximately 54 months. Researchers will track the time to first event of any component of MACE-PLUS and collect data to assess the effect of AZD0780 compared to placebo. The study includes regular visits and evaluations to ensure participant safety and adherence to treatment.

Researchers are investigating how plant-based diets might change the gut microbiome and increase the production of beneficial short-chain fatty acids (SCFAs) in patients with smoldering multiple myeloma (sMM). The goal is to see if these dietary changes can support a healthier immune balance and potentially prevent progression to active multiple myeloma. This national, prospective, multicenter study will include up to 62 sMM patients and focuses on understanding how diet impacts gut bacteria and immune cell ratios in this population. Participants who qualify and enroll will be encouraged to follow a balanced, high-fiber diet for 12 weeks, choosing from various diet plans or individual foods based on their preferences. Nutritional counseling will be provided at enrollment, one month later, and as needed. Medical visits will be free and conducted at enrollment and week 12. Patients will be asked to track their daily diet choices through questionnaires throughout the study. Stool and blood samples will be collected at baseline, 4 weeks, and 12 weeks to assess changes in gut microbiota and immune markers. Study participants will undergo routine clinical follow-up that includes bone marrow and blood sample collection at screening, as well as stool sampling at the start, 4 weeks, and 12 weeks after beginning the diet. Researchers will measure the mean SCFA concentration in stool samples to evaluate the diet's effects. Dietary adherence will be monitored via questionnaires. The overall study duration for each participant is 12 weeks, during which their health and immune response will be closely observed to understand the impact of the plant-based diet.

Researchers are conducting a Phase 3 study to compare two front-line treatments for adults with nonsquamous non-small cell lung cancer (NSCLC) that is stage IV or advanced stage IIIB/C. The study focuses on patients whose tumors have a KRAS p.G12C mutation and are negative for PD-L1 expression. The main goal is to evaluate how each treatment affects progression-free survival and overall survival over about 2.5 years. Participants will be randomly assigned to receive either sotorasib combined with platinum doublet chemotherapy or pembrolizumab combined with platinum doublet chemotherapy. Sotorasib is given orally, while pembrolizumab is given intravenously. Both groups will receive the combination therapies as their initial treatment for advanced NSCLC. During the study, participants will be monitored regularly to assess treatment effects and safety. Researchers will track how long patients live without the cancer worsening and overall survival over approximately 2.5 years. The study includes evaluations to determine eligibility and ongoing assessments to monitor health and treatment response throughout the trial period.

Researchers are evaluating the effectiveness, safety, and tolerability of a combination treatment including adagrasib, pembrolizumab, and platinum-doublet chemotherapy compared to a placebo combined with pembrolizumab and platinum-doublet chemotherapy. This study focuses on adults with previously untreated, locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) that has a KRAS G12C mutation. The trial is a randomized, double-blind, phase 3 study designed to provide insights into treatment options for this specific lung cancer type. Participants receive either adagrasib plus pembrolizumab alongside platinum-doublet chemotherapy drugs such as carboplatin or cisplatin and pemetrexed, or they receive a placebo plus pembrolizumab and the same chemotherapy regimen. The dosages and schedules of these drugs are specified and administered on predetermined days. The trial compares these two treatment groups to understand better the impact of adding adagrasib to the existing pembrolizumab and chemotherapy treatment. Throughout the study, participants are closely monitored for progression-free survival and overall survival, assessed up to seven years using standardized criteria for tumor response. Regular imaging scans such as CT or MRI are used to measure disease status. Safety and tolerability are also evaluated during the study, with ongoing assessments to track adverse effects and treatment response. The total duration of follow-up allows for long-term observation of treatment outcomes and participant health.

Researchers are evaluating the safety and effectiveness of Ifinatamab Deruxtecan (I-DXd) compared to treatment chosen by physicians for adults with relapsed extensive-stage small cell lung cancer (ES-SCLC). The study aims to find out if I-DXd can improve the objective response rate, meaning the proportion of patients whose cancer shrinks or disappears, and extend overall survival time compared to other treatments. Secondary goals include assessing safety, patient-reported outcomes, immune response to I-DXd, B7-H3 protein levels, and how the drug is processed in the body. Participants will receive either I-DXd at a dose of 12 mg/kg given intravenously on the first day of each 21-day treatment cycle or one of the physician's choice treatments including Topotecan, Amrubicin, or Lurbinectedin, administered according to local standards of care. The study is randomized and open-label, meaning treatments are assigned by chance and both patients and doctors know which treatment is given. During the study, participants will be closely monitored with tumor assessments to evaluate response and detect disease progression, safety evaluations, and quality of life questionnaires. The main outcomes measured are the objective response rate assessed by a blinded independent review and overall survival time, tracked for up to approximately five years after randomization. Researchers will also monitor for any adverse effects and collect health economics data to understand the broader impact of treatments.