San Rafael

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.

Researchers are evaluating the efficacy, safety, and pharmacokinetics of INM004 in children with Hemolytic Uremic Syndrome caused by Shiga toxin-producing Escherichia coli STEC-HUS. This study aims to assess how well INM004, when added to standard care, improves kidney function and reduces complications, mortality, and hospital stay in affected pediatric patients. Participants will be randomly assigned to receive either two doses of INM004, an Anti-Shiga Toxin Hyperimmune Equine Immunoglobulin fragment, or two doses of a placebo solution. Each dose is given intravenously 24 hours apart, with infusion times adjusted based on body mass index. The study includes a 28-day acute phase and further assessments up to 90 days to monitor recovery and other health outcomes. During the study, children will undergo evaluations of kidney function, blood tests for markers of disease activity, and safety assessments. Researchers will measure the time to kidney function recovery as the primary outcome, along with mortality rates, dialysis needs, and hospital stay length. The trial lasts for at least 90 days, including close monitoring of health status and medication effects throughout this period.