Neuquen

Researchers are evaluating the effectiveness, safety, and how the body processes INM004 in children with Hemolytic Uremic Syndrome caused by infection with Shiga toxin-producing Escherichia coli (STEC-HUS). The study aims to see if adding INM004 to the usual treatment helps improve kidney function and reduce complications, mortality, and hospital stay time. This is a Phase III study focusing on pediatric patients with this condition. Participants will receive either INM004 or a placebo, both given as two intravenous infusions 24 hours apart. INM004 involves doses of Anti-Shiga Toxin Hyperimmune Equine Immunoglobulin F(ab´)2 fragments calculated by the child's weight, infused over 50 minutes or 100 minutes for those with a body mass index over 30. The placebo matches the infusion schedule and appearance but contains no active drug. During the study, participants will be closely monitored for kidney function recovery over 28 days and other health outcomes. Assessments include laboratory tests for blood and kidney function, safety evaluations, and pharmacokinetics of INM004. Hospitalization will occur at the participating institution, with informed consent and pregnancy testing as part of the process. The study includes children from 9 months to under 18 years old and lasts through the acute phase recovery period.

Researchers are tracking patients with Fabry disease through an ongoing international, multi-center observational program called the Fabry Registry. This program collects routine clinical data from patients regardless of their treatment status to better understand the disease's variability, progression, and natural history. It also focuses on enhancing patient care by supporting the development of monitoring recommendations and evaluating the long-term safety and effectiveness of Fabrazyme, a treatment for Fabry disease. The study includes a Fabry Pregnancy Sub-registry, which is a voluntary, international, longitudinal observation program that monitors pregnancy outcomes for women enrolled in the Fabry Registry who are pregnant or have been pregnant. This sub-registry collects medical and obstetric history, pregnancy, and birth data, along with infant growth information up to 36 months postpartum, regardless of the specific treatment received. No experimental treatments are administered in either registry; patients continue receiving routine care as determined by their physicians. Participants contribute data through clinical assessments and standard care evaluations performed by their doctors. The study measures long-term outcomes including safety and effectiveness of Fabrazyme over up to 33 years, as well as pregnancy outcomes and infant growth data. The program helps fulfill regulatory requirements and supports research while tracking patient health over extended periods without altering their usual care.