An open-label, single-arm, multicenter study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy without first-line therapeutic plasma exchange in adults with immunemediated thrombotic thrombocytopenic purpura

This study is about finding a new way to treat a rare but serious blood disease called immune-mediated Thrombotic Thrombocytopenic Purpura, or iTTP for short. In iTTP, your body mistakenly attacks itself, leading to tiny blood clots forming in small blood vessels. This can cause problems with your blood, kidneys, brain, and heart.

Currently, a common treatment for iTTP is called therapeutic plasma exchange, often shortened to TPE. This is a procedure where a machine filters your blood to remove harmful substances. While TPE is effective, it can be quite intensive and involves frequent hospital visits. This study is exploring if a new medicine called Cablivi, given along with other medicines that calm down your immune system, can help adult patients with iTTP get better and go into remission without needing the TPE procedure.

The main goal of the study is to see how many patients get better and into remission without needing TPE. The researchers will also be carefully watching for any side effects and how quickly people respond to the new treatment. This is an important step in potentially offering patients with iTTP a different, possibly less intensive, treatment option in the future.