This study investigates the effects of an investigational medication compared to an existing treatment for Gaucher disease Type 3 (GD3). Gaucher disease is a genetic disorder where fatty substances accumulate in certain organs. This study aims to evaluate if the investigational medication can stabilize or improve neurological symptoms and maintain …
This study investigates the safety and effects of a single dose of an investigational medication in adults with osteogenesis imperfecta (OI). Osteogenesis imperfecta is a genetic disorder characterized by fragile bones that break easily. The purpose of this study is to assess how the investigational medication is tolerated and how …
This study investigates the safety and efficacy of an investigational gene therapy for adults with phenylketonuria (PKU). PKU is an inherited disorder caused by issues with the gene coding for the phenylalanine hydroxylase (PAH) enzyme, leading to high levels of phenylalanine (Phe) in the blood. If untreated, high Phe levels …
This study investigates an investigational medication for people with moderate to severe Ulcerative Colitis (UC). UC is a long-term condition that causes inflammation and sores in the colon. The purpose of this study is to understand how well the investigational medication works and how safe it is compared to a …
This study investigates bronchiectasis, a long-lasting lung disease that causes the air passages in the lungs to enlarge due to weakened airway walls. Symptoms include shortness of breath, coughing up mucus or blood, wheezing, and frequent lung infections. The purpose of this study is to explore an investigational medication that …
Who can participate: Participants aged 18 to 65 years with a confirmed diagnosis of Condition X are eligible. Key eligibility factors include stable health status and no prior treatment with similar investigational drugs.Study details: Participants will receive either the investigational treatment or a placebo. The trial will involve regular monitoring …
This study investigates the effects of vagal nerve stimulation during sinus surgery. The purpose is to see if this method can reduce bleeding and improve the view for surgeons. Functional endoscopic sinus surgery (FESS) is a procedure used to treat sinus issues. During this surgery, bleeding can make it hard …
This study investigates an investigational treatment for individuals under the age of 18. The purpose of this study is to understand the effects and safety of this treatment in young participants.Participants in this study will undergo various procedures to monitor their health and the effects of the treatment. These procedures …
This study investigates the safety and immune response of investigational vaccines for meningococcal disease, specifically targeting serogroup B, in healthy individuals across various age groups. The purpose is to compare these investigational vaccines with two licensed vaccines, focusing on their effects when given alone or with other routine vaccines. Meningococcal …
This study investigates the antibody response to a meningococcal vaccine in infants and toddlers aged 6 weeks to 18 months. The purpose is to compare the response to a 3-dose series of an investigational meningococcal vaccine with a licensed vaccine, both given alongside routine pediatric vaccines. Meningococcal disease is a …
