This study investigates the use of an investigational gene therapy for adults with phenylketonuria (PKU), a condition where the body cannot break down an amino acid called phenylalanine (Phe). The purpose of this study is to see if the investigational treatment can safely reduce Phe levels and allow participants to …
This study investigates the effects of an investigational medication for people with moderate-to-severe atopic dermatitis (AD). Atopic dermatitis is a skin condition that causes itchy, inflamed skin. The study focuses on participants aged 12 years and older who have not responded well to previous treatments, including biologic medications or oral …
This study investigates the effects of an investigational medication on adults with non-responsive celiac disease (NRCD) who are on a gluten-free diet. Celiac disease is a condition where eating gluten leads to damage in the small intestine. The study aims to see how the investigational medication affects the small intestine's …
This study investigates the efficacy and safety of an investigational medication in delaying disability progression in individuals with non-relapsing secondary progressive multiple sclerosis (nrSPMS). nrSPMS is a stage of multiple sclerosis (MS) where symptoms worsen over time without periods of remission. Participants will be randomly assigned to receive either the …
This study investigates the effects of an investigational medication compared to an existing treatment in people with relapsing forms of multiple sclerosis, a condition where the immune system mistakenly attacks the protective covering of the nerves, causing communication problems between the brain and the rest of the body. The study …
This study investigates the treatment of newly diagnosed moderate or severe chronic graft-versus-host disease (cGVHD). Chronic GVHD is a condition that can occur after a stem cell or bone marrow transplant, where the donated cells attack the recipient's body. The study aims to assess the effectiveness of an investigational medication …
This study investigates the effectiveness and safety of an investigational medication compared to a placebo in adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) who do not respond to available treatments. CIDP is a condition where the body's immune system mistakenly attacks the protective covering of the nerves, leading to weakness …
This study investigates the effectiveness and safety of an investigational medication compared to intravenous immunoglobulin (IVIg) in adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). CIDP is a neurological disorder that causes weakness and sensory loss due to inflammation of the peripheral nerves. The purpose of this study is to assess …
This study investigates a meningococcal vaccine in children under 10 years old. Meningococcal disease is a serious illness caused by bacteria that can lead to infections in the brain and spinal cord. The purpose of this study is to compare two formulations of a new investigational vaccine with existing vaccines. …
This study investigates the effectiveness of a treatment for adults with relapsed or refractory multiple myeloma, a type of blood cancer. Multiple myeloma affects plasma cells, which are a type of white blood cell. The study looks at how well the investigational medication works when given under the skin along …