This study investigates the use of a subcutaneous investigational medication in combination with carfilzomib and dexamethasone for adults with relapsed or refractory multiple myeloma. The study aims to determine how effective this combination is in treating this condition.Participants in the study will receive the investigational medication through a system that …
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 effectiveness of an investigational gene therapy in individuals with non-congenital Myotonic Dystrophy Type 1 (DM1). The purpose is to assess how well the treatment reduces levels of a specific protein that affects muscle function.Participants will receive a single intravenous dose of the investigational gene …
This study investigates the effectiveness and safety of an investigational medication in adults with immune thrombocytopenia (ITP) who did not respond to first-line treatment.Participants in this study will take the investigational medication orally. The study includes several phases: a screening period, a primary analysis period, a long-term extension for some …
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 the safety and efficacy of a one-time intravitreal injection of an investigational medication for participants with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). The study is divided into two parts to assess different aspects of the treatment.Participants will receive a one-time injection into the eye, …
This study investigates the clinical outcomes of patients with Gaucher disease through an observational registry. The purpose of the registry is to gather data on how the disease progresses and affects patients over time, without altering their treatment plans. This helps in understanding the natural history of the disease and …
This study investigates Mucopolysaccharidosis I (MPS I), a rare genetic disorder. The purpose of this observational study is to collect data on the natural history and progression of MPS I and to observe clinical responses in patients receiving enzyme replacement therapy or other treatments.Participants in this study will have their …
This study investigates Pompe disease, a rare genetic disorder. The purpose of this observational study is to track the disease's natural history and outcomes in patients, both those receiving treatment and those who are not. An observational study is a type of research study where data is collected by observing …
This study investigates Fabry disease, a rare genetic disorder. The purpose of this study is to track the routine clinical outcomes for patients with Fabry disease, without altering their treatment. This is an observational study, meaning that data is collected by observing participants receiving routine care, without changing their treatment, …
