This study investigates an investigational medication for treating infantile-onset Pompe disease (IOPD). IOPD is a rare genetic disorder where the body lacks an enzyme called acid alpha-glucosidase, which helps break down glycogen, a type of sugar stored in muscles. This can lead to muscle weakness, heart problems, and other health …
This study investigates the effects of an investigational medication in people with primary progressive multiple sclerosis (PPMS). PPMS is a type of multiple sclerosis (MS) that causes a gradual worsening of symptoms without relapses. The aim is to see if the investigational medication can delay the progression of disability compared …
This study investigates an investigational medication combined with other agents to treat relapsed or refractory myeloma, a type of blood cancer. Myeloma affects plasma cells in the bone marrow and can cause bone pain, fatigue, and increased risk of infections. The study includes multiple substudies to explore different combinations and …
This study investigates the use of an investigational medication in people who have received or will receive a kidney transplant. Kidney transplantation is a procedure where a healthy kidney from a donor is placed into a person whose kidneys are not working properly. The study focuses on two groups, or …
This study investigates an investigational medication for adults with moderate to severe atopic dermatitis (AD). Atopic dermatitis is a condition that causes red, itchy skin. This study aims to understand the safety and effectiveness of the investigational medication in participants who have already been part of a previous trial involving …
This study investigates the safety and effectiveness of an investigational medication given alone or with other anti-cancer treatments. It is for adults aged 18 and older who have metastatic cancers that have been treated before. Metastatic cancer means the cancer has spread from where it started to other parts of …
This study investigates the use of caplacizumab and immunosuppressive therapy for treating immune-mediated thrombotic thrombocytopenic purpura (iTTP). iTTP is a condition where the immune system mistakenly attacks certain proteins in the blood, leading to low platelet levels. This can cause problems like easy bruising and bleeding. The purpose of this …
This study investigates the effects of an investigational medication on adults with moderately to severely active Ulcerative Colitis (UC) who have an eosinophilic phenotype. Ulcerative Colitis is a chronic condition where the colon and rectum become inflamed. The study aims to understand how well the investigational medication works and its …
This study investigates the long-term safety and effects of an investigational medication in people aged 12 and older with moderate to severe atopic dermatitis (AD). Atopic dermatitis, also known as eczema, is a condition that makes the skin red, inflamed, and itchy. The purpose of this study is to understand …
This study investigates the treatment of moderate to severe atopic dermatitis (AD), a skin condition causing itchy, inflamed skin. The purpose is to assess how effective and safe a solution for injection is compared to a placebo in people aged 12 and older with AD that is not well controlled …
