Neuquen

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.

Researchers are tracking patients with Fabry disease through an ongoing international observational program called the Fabry Registry. This registry collects routine clinical outcomes for patients regardless of whether they are receiving treatment. The study aims to better understand the diseases variability, progression, and natural history, including in women who carry one copy of the gene, and to help improve patient care by developing monitoring recommendations and reporting outcomes. Additionally, the registry evaluates the long-term safety and effectiveness of Fabrazyme4, a treatment used in Fabry disease. The registry includes a special pregnancy sub-registry for women with Fabry disease who are pregnant or have been pregnant. This sub-registry observes pregnancy outcomes and infant growth up to 36 months after birth, collecting medical and obstetric history and treatment details. No experimental treatments are given participants continue to receive their usual care as determined by their physicians. Data from both registries support regulatory requirements and ongoing research. Participants undergo regular clinical assessments and receive standard care from their doctors throughout the study. The research team collects data on disease progression, treatment effectiveness, pregnancy outcomes, and infant development. The study is observational, meaning no study drugs or procedures are administered. The total participation can last up to 33 years, allowing for long-term monitoring of safety and outcomes related to Fabry disease and pregnancy.