This study investigates the effects of switching from emicizumab to an investigational medication for individuals with severe hemophilia A. The purpose of this study is to understand the safety and tolerability of the investigational medication after participants transition from their current preventive treatment.Participants will undergo a screening period followed by …
This study investigates the use of an investigational medication in combination with other treatments for adults who have relapsed or refractory multiple myeloma.The study focuses on evaluating the effectiveness of this combination treatment. Researchers aim to understand how well the treatment works in improving the condition of participants who have …
This study investigates the effectiveness of different investigational medications compared to a placebo in improving lung capacity in participants with Interstitial Lung Disease (ILD) secondary to Systemic Sclerosis (SSc). The purpose is to observe changes from the start to the end of the treatment period at Week 52.Participants in the …
This study investigates the safety and effects of an investigational mRNA vaccine for individuals with mild acne. The study will include two groups, a Sentinel Cohort to initially assess safety, and a Main Cohort for broader evaluation.Participants will receive two administrations of the investigational vaccine. The study will consist of …
This study investigates a treatment for geographic atrophy, a condition related to age-related macular degeneration (AMD). The purpose of this study is to assess the safety and effectiveness of a one-time injection in the eye for people with geographic atrophy. The study will look at how well the treatment works …
This study investigates the safety and ability of an investigational pneumococcal vaccine to help the body develop antibodies in children and adolescents with sickle cell disease. The study is focused on children aged 2 to 17 years, who may or may not have received previous vaccinations against pneumococcal disease.The study …
This study investigates the experiences of adults with relapsed or refractory multiple myeloma. The purpose is to gather data about patients receiving standard care with an investigational medication. Researchers aim to collect various types of data, including quantitative, qualitative, and wearable data, to understand the treatment experience better.Participants will be …
This study investigates the administration of an investigational medication for people with multiple sclerosis (MS). The purpose is to compare two ways of giving the medication: under the skin (subcutaneous) and into the vein (intravenous).The study focuses on understanding how these two methods of administration affect the treatment of MS. …
This study investigates Gaucher disease, a genetic disorder characterized by the accumulation of Gaucher cells in areas like the spleen, liver, and bone marrow. The purpose of this study is to observe patients with Gaucher disease to better understand the condition and its progression.The study focuses on understanding the variability …
This study investigates Pompe disease, focusing on gathering information about how the disease progresses over time in patients who are both treated and untreated.The study focuses on understanding the variability and natural history of Pompe disease. It aims to provide insights that can help improve patient care and evaluate the …
