Elqui

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.

Researchers are evaluating whether providing tuberculosis preventive treatment (TPT) during the "window period" can reduce new tuberculosis infections in children and adolescents who live with someone recently diagnosed with pulmonary tuberculosis. This cluster-randomized controlled trial focuses on preventing new infections by comparing immediate TPT to standard care, which only offers treatment to those already testing positive for infection. The study targets children and adolescents aged 5 to under 18 years who are household contacts of tuberculosis patients. Participants will be assigned to one of two groups. One group receives weekly doses of isoniazid and rifapentine for 12 weeks regardless of infection status at the start, while the other group receives the same treatment only if they test positive for infection at baseline. The treatment involves a 12-week course of the 3HP regimen, taken weekly. Those in the control group who test negative initially but convert to positive during follow-up also receive treatment after the 12-week period. Throughout the study, participants will undergo testing with the Interferon-Gamma Release Assay (IGRA) at enrollment and again after 12 weeks to detect new tuberculosis infections. Researchers will track the number of new infections by measuring IGRA conversions over this period. The study includes monitoring for treatment adherence and safety, with follow-up lasting at least 12 weeks. The results aim to inform better prevention strategies for tuberculosis in children and adolescents exposed in household settings.