Gallarate

Researchers are evaluating the efficacy and safety of remibrutinib in patients with secondary progressive multiple sclerosis SPMS. This is a Phase III, randomized, double-blind, placebo-controlled, multi-center study involving approximately 1275 participants. The study aims to provide important data on remibrutinibs effect on disability progression in SPMS and includes both a Core Part and an Extension Part for further assessment. Participants are randomly assigned to receive either remibrutinib or a matching placebo as oral film-coated tablets during the Core Part. The Core Part includes double-blind treatment, followed by an Extension Part where all participants receive open-label remibrutinib tablets. Treatment is taken orally, and the study is event-driven, continuing until required endpoints are met. During the study, participants undergo regular assessments of disability progression using the Expanded Disability Status Scale EDSS, Timed 25-Foot Walk, 9-Hole Peg Test, and Symbol Digit Modalities Test, among others. Brain imaging and safety monitoring for adverse events are performed throughout up to approximately five years. Researchers track changes in brain lesions and atrophy, and follow participants for safety and treatment effects over time.

Primary biliary cholangitis PBC is a rare autoimmune liver disease that causes chronic inflammation of the bile ducts, leading to serious liver damage if untreated. This research aims to create a national database of PBC patients in Italy linked to biological sample storage to improve understanding of the disease, especially in patients who do not respond well to current treatment. The study involves about 60 centers across Italy and focuses on the biology of PBC and disease progression. Participants will have their clinical and laboratory data collected at the start and then annually through electronic case report forms. Biological samples will also be gathered to study patients genes and immune system differences among various PBC subtypes. This observational study collects data from adult PBC patients diagnosed based on established clinical, antibody, or histological criteria. Throughout the 10-year study, researchers will analyze patient phenotypes, responses to ursodeoxycholic acid therapy, factors affecting disease progression, and the safety and effectiveness of new treatments. Participants will be monitored regularly, with their clinical profiles and biological data helping to support future research, clinical care, and drug development efforts for PBC.

Primary sclerosing cholangitis PSC is a rare, chronic liver disease marked by inflammation and scarring of bile ducts, leading to liver failure and increased risk of colorectal and liver cancers. Both children and adults can be affected, with a median survival of 17 years after diagnosis. PSC is a significant cause for liver transplantation in some countries, and it is closely linked to inflammatory bowel disease. This research aims to develop Italys first national PSC patient database linked with biological sample storage to better understand the diseases prevalence and characteristics. This observational study involves recruiting PSC patients across Italy aged 17 and older. Clinical and laboratory data will be collected at baseline and annually using electronic case report forms. Biological samples will also be stored to support research into patient profiles, disease progression, biomarkers, and therapy responses. The study will run for 10 years to assess long-term safety and effectiveness of new treatments and to identify patient subtypes at higher risk. Participants will provide clinical information and biological samples during their involvement. Researchers will monitor disease progression, therapy responses, and safety data over a decade. The main goal is to identify distinct patient subgroups with higher risks and to collect detailed clinical and laboratory data to improve understanding of PSC. This long-term follow-up aims to guide future treatment and management strategies for PSC patients in Italy.