Cagliari

Researchers are studying multiple myeloma MM, a complex blood cancer, to improve how well doctors can predict which patients might experience early disease return after treatment. This research uses advanced machine learning methods to analyze clinical, genomic, and imaging information from MM patients, aiming to develop models that better forecast treatment response and risk of early relapse. The study is observational and conducted across multiple centers with a focus on improving personalized care for MM patients. The study collects and processes a wide range of data from patients with active multiple myeloma, including clinical details, tumor characteristics, and immune markers. Machine learning tools will combine this data to identify patient groups with similar disease features related to early progression. There are no experimental treatments given, as this is a non-interventional study that observes and analyzes existing patient data to enhance risk prediction models. Participants aged 18 years or older with a diagnosis of active multiple myeloma who agree to join the study will have their clinical and biological data collected and analyzed. The main outcome measured is the overall response rate to therapy within 12 months from the start of treatment. This study aims to generate new insights to support personalized treatment decisions and better management of MM. The study starts in October 2024 and will continue until August 2026, with no additional treatments or procedures beyond regular care and data collection.

Researchers are investigating the effectiveness and safety of combining petosemtamab with pembrolizumab compared to pembrolizumab alone as first-line treatment for recurrent or metastatic PD-L1 positive head and neck squamous cell carcinoma HNSCC. This Phase 3 randomized, open-label study focuses on patients who have not received previous systemic therapy for incurable recurrent or metastatic disease and aims to improve treatment outcomes in this population. Participants will be randomly assigned to receive either the combination of petosemtamab plus pembrolizumab or pembrolizumab alone. The study excludes patients previously treated with anti PD-L1 or anti-EGFR therapies, with some exceptions for local treatments like cetuximab under specific conditions. The treatments will be administered as part of the trial, with researchers comparing the two approaches over the treatment period. During the study, participants will undergo regular evaluations including tumor assessments based on RECIST v1.1 criteria, health-related quality of life questionnaires, and safety monitoring for adverse events. The main outcomes measured include overall survival and objective response rate up to approximately three years. Additional assessments include progression-free survival, duration of response, and pharmacokinetics. These activities aim to provide comprehensive data on how well the treatments work and their safety profiles throughout the study period.

Researchers are assessing the effectiveness and safety of rilvegostomig combined with fluoropyrimidine and trastuzumab deruxtecan compared to trastuzumab, chemotherapy, and pembrolizumab in adults with HER2-positive locally advanced or metastatic gastric or gastroesophageal junction GEJ adenocarcinoma whose tumors express PD-L1 CPS 1. The study also evaluates rilvegostomig combined with trastuzumab and chemotherapy to understand the contribution of each treatment component. This is a Phase 2, randomized, open-label, global, multicenter trial sponsored by AstraZeneca. Participants are divided into three groups Arm A receives T-DXd, rilvegostomig, and fluoropyrimidine capecitabine or 5-FU Arm B receives pembrolizumab, trastuzumab, and chemotherapy either 5-FU plus cisplatin or capecitabine plus oxaliplatin Arm C receives rilvegostomig, trastuzumab, and chemotherapy 5-FU plus cisplatin or capecitabine plus oxaliplatin. Treatments are given by intravenous infusion every three weeks or oral administration twice daily for capecitabine. This setup allows comparison of different combinations to evaluate each drugs role. During the study, participants will be monitored for progression-free survival and overall survival up to about six years. Researchers will also assess response rates, duration of response, adverse events, pharmacokinetics, immunogenicity, and quality-of-life factors like eating difficulties and side-effect burden. The study involves regular assessments including tumor measurements and laboratory tests. Participation may last several years, with safety and efficacy closely followed throughout this time.

Researchers are conducting a large prospective, observational cohort study to evaluate the clinical impact of new Monoclonal Antibodies MAB in patients with B-cell Non-Hodgkin Lymphoma NHL treated in Italian clinical practice. The study focuses on collecting information about the use, feasibility, effectiveness, and both short- and long-term side effects of novel MABs that have been approved by the European Medicines Agency since 2020 and prescribed according to authorized indications in Italy. Participants will be divided into groups based on treatment indication, antibody type, and lymphoma subtype to allow detailed analysis. The study observes patients with B-cell NHL who have received at least one dose of a novel MAB either alone or in combination, as authorized for use in Italy. Both patients receiving first-line treatment and those with relapsed or refractory disease are included. Various cohorts and sub-cohorts will be analyzed to understand outcomes by indication, antibody type, and histological subtype, providing a comprehensive overview of these treatments in real-life settings. Participants will be followed for at least five years to assess outcomes such as overall response rate, complete response rate, progression-free survival, overall survival, event-free survival, time to next treatment, non-relapse mortality, duration of response, and the incidence of early and late adverse events. Clinical data collection will include treatment details, safety monitoring, and long-term follow-up to evaluate both effectiveness and toxicity. The study spans several years, allowing researchers to capture extensive real-world evidence on novel MAB use in B-cell NHL.

This research aims to observe the real-world use and effects of pegcetacoplan in adults diagnosed with Paroxysmal Nocturnal Hemoglobinuria PNH. As a new treatment with a unique mechanism of action, pegcetacoplans effectiveness and safety in routine medical practice are being studied to provide valuable information for patients, healthcare providers, and payers. The study will also gather data on blood transfusions and healthcare resource use before and after starting pegcetacoplan. Patients who have started pegcetacoplan treatment within the past 12 months or are prescribed the drug at enrollment will be included. Data collection includes retrospective information from up to 12 months before treatment start and prospective monitoring for approximately 36 months, with the total data period extending up to about 48 months. After stopping pegcetacoplan, patients remain in the study for 8 weeks to record any adverse events. Patients continue regular clinic visits, where data from each visit will be gathered. Participants will have data collected on various health measures such as hemoglobin levels, blood markers, transfusion needs, and patient and physician treatment satisfaction at regular intervals up to 36 months. Safety and adverse events will be monitored throughout. The main outcome measured is the change in hemoglobin level from treatment start to 6 months. This long-term observational study allows for comprehensive tracking of pegcetacoplans use and effects over time in usual care settings.

Researchers are evaluating whether baricitinib can delay the development of clinical stage 3 type 1 diabetes T1D in children and adults at high risk. This phase 3, double-blind, randomized, placebo-controlled study includes participants aged 1 to under 36 years who have early stages of T1D or specific diabetes-related autoantibodies. The study aims to better understand preventing or delaying the onset of clinical diabetes in this at-risk population. Participants will be randomly assigned to receive either baricitinib or a placebo orally. The study lasts up to approximately 5 years, during which participants take the assigned treatment and are monitored regularly. The trial includes a control group receiving placebo to compare with the baricitinib group. The dosing schedule and exact treatment duration depend on the study protocol and participant response. Throughout the study, participants will undergo assessments including monitoring the time to diagnosis of stage 3 T1D, blood tests measuring glucose and C-peptide levels, body measurements, and health surveys. Researchers will track changes in these measures over time, including pharmacokinetics of baricitinib. Safety and response to treatment will be closely observed throughout the study duration to evaluate the effects of the medication.

Researchers are evaluating whether eptinezumab can reduce the number of days with episodic migraine in children and adolescents aged 6 to 17 years. This Phase 3 clinical trial focuses on the preventive treatment of episodic migraine in pediatric participants using intravenous infusion. The study is randomized, double-blind, and placebo-controlled to compare the effects of different doses of eptinezumab with a placebo. Participants will be assigned to one of three groups a single intravenous infusion of eptinezumab 300 mg adjusted for weight, a single intravenous infusion of eptinezumab 100 mg adjusted for weight, or a single intravenous infusion of a placebo matching eptinezumab. The dosing occurs once, and the study monitors results over subsequent weeks. This design allows for comparison of the medication against placebo to evaluate treatment effects in this population. During the study, participants and their caregivers will complete headache diaries to document migraine and headache days. Researchers will assess changes in the number of monthly migraine days over 12 weeks, response rates to treatment, headache duration and intensity, acute medication use, and disability related to migraine using questionnaires like the Pediatric Migraine Disability Assessment PedMIDAS. Blood samples will be collected to measure drug concentrations and antibodies. Safety and efficacy are monitored throughout the study period, which concludes approximately 12 weeks after dosing.

Researchers are evaluating icotrokinra for its effectiveness and safety in people with moderately to severely active Crohns disease, a condition causing severe inflammation in the intestines. This clinical trial is a Phase 2b3 study aiming to understand how well icotrokinra works compared to placebo to improve symptoms and intestinal healing. Participants will be randomly assigned to receive one of several treatments two different doses of icotrokinra or a matching placebo, taken orally every day during an induction period of up to 12 weeks. Based on their response at Week 12, participants may continue with maintenance dosing or placebo up to Week 40. Those completing the maintenance phase may join a long-term extension study for further evaluation. During the trial, participants will be monitored with clinical assessments, endoscopy, and patient-reported outcomes to measure response, remission, and safety. The main outcomes include clinical response and remission at Weeks 12 and 40, along with endoscopic healing. Safety will be tracked through adverse event reporting up to four weeks after the last dose. The study is expected to continue until 2032, with multiple visits for treatment and evaluations throughout.

Researchers are evaluating the efficacy and safety of belimumab compared to a placebo, both given alongside standard therapy, for adults with systemic sclerosis associated interstitial lung disease SSc-ILD. The study aims to assess how belimumab affects lung function, skin thickening, and symptoms like fatigue that influence quality of life in these patients. This is a Phase 23 randomized, double-blind, placebo-controlled trial sponsored by GlaxoSmithKline. Participants will be randomly assigned to receive either belimumab or a placebo, both administered subcutaneously along with standard therapy. The treatment period lasts 52 weeks, during which the effects on lung function and skin symptoms will be closely monitored. The study involves two groups one receiving belimumab plus standard care and the other receiving placebo plus standard care. During the study, participants will undergo assessments including lung function tests such as Forced Vital Capacity FVC, skin thickness scoring, fatigue evaluations, and quality of life questionnaires at baseline and Week 52. Researchers will also track any adverse events and serious side effects. The main outcome measured is the change in lung function after 52 weeks. Participants will be monitored regularly throughout the 52-week treatment period to evaluate treatment effects and safety.

This research focuses on adults with moderate-to-severe Crohns Disease CD, a chronic inflammatory condition of the digestive tract. It aims to evaluate how effective two approved drugs, upadacitinib and risankizumab, are as second-line treatments for CD in real-world clinical settings. Approximately 250 participants prescribed either drug by their physicians will be enrolled across 25 sites in Italy. Participants will receive either upadacitinib or risankizumab as part of their usual medical care, following local prescribing guidelines. The study includes a retrospective phase looking back up to 2 years before enrollment and a prospective phase where participants will be observed for up to 18 months after starting the medication. Both groups will be followed from their first dose throughout the study period. During this time, participants will not experience any additional study-related burden beyond their routine care. Researchers will review clinical records and monitor outcomes such as the percentage of participants achieving corticosteroid-free remission at 12 months. The study aims to gather real-world data on these treatments effectiveness and safety over time, with no extra visits or procedures required beyond standard practice.