Ponta Delgada

Researchers are tracking the natural history and clinical outcomes of patients with Gaucher disease through the ICGG Gaucher Registry, an international, multi-center observational program. This registry does not involve any experimental treatments, but collects information to better understand the variability, progression, and identification of Gaucher disease, aiming to improve patient care and therapeutic guidance. It also evaluates the long-term use of treatments like imiglucerase and eliglustat. The study includes two groups patients with Gaucher disease who receive routine clinical assessments and standard care as determined by their physicians, and a Pregnancy Sub-registry for women with Gaucher disease who are pregnant or have been pregnant. The Pregnancy Sub-registry collects information on pregnancy outcomes, complications, and infant growth up to 36 months postpartum, regardless of whether the women receive disease-specific therapy. Participants undergo clinical assessments and receive care according to their treating physicians decisions. Data collected includes medical history, pregnancy and birth details for the sub-registry, and patient outcomes over time. The primary goals are to provide recommendations for monitoring Gaucher disease patients, report outcomes to optimize care, and track pregnancy and infant growth outcomes. This ongoing registry allows long-term follow-up without experimental interventions.

Researchers are evaluating how well seltorexant works and its safety when used alongside antidepressants in adults and elderly people with major depressive disorder and insomnia symptoms. This study focuses on participants who have not had adequate improvement from their current antidepressant treatment with selective serotonin reuptake inhibitors SSRIs or serotonin-norepinephrine reuptake inhibitors SNRIs. The trial aims to understand both the initial effects and the longer-term maintenance of seltorexant compared to placebo in this population. Participants in the study will receive either seltorexant or a matching placebo orally once daily for six weeks during the first double-blind treatment phase, while continuing their baseline SSRI or SNRI antidepressant. Those who complete Part 1 and meet criteria will enter Part 2, which includes an open-label phase where all receive seltorexant alongside their antidepressant. Participants achieving stable response may then continue with seltorexant or placebo during the double-blind maintenance phase. Throughout, all continue their original antidepressant treatment. During the study, participants will undergo assessments including depression rating scales such as the Montgomery-Asberg Depression Rating Scale MADRS and sleep disturbance questionnaires like the PROMIS-SD. The main outcomes include changes in depression scores after 43 days and time to relapse during maintenance, which may last up to nearly three years. Safety and tolerability will be closely monitored, and participants who do not continue to Part 2 will have a post-treatment follow-up after Part 1. Total participation may extend over several months or years depending on the study phase.