Observational Study to Assess Safety and Immune Response to Adzynma in Children and Adults with Congenital Thrombotic Thrombocytopenic Purpura cTTP

What this Trial Is About

Congenital thrombotic thrombocytopenic purpura cTTP is a rare inherited blood disorder caused by a genetic problem that affects the bodys ability to produce an enzyme called ADAMTS13. This enzyme normally helps break down a larger protein called von Willebrand Factor vWF. People with cTTP have low ADAMTS13 levels, leading to buildup of large vWF forms that cause blood clots in small vessels, potentially blocking blood flow to vital organs. Researchers are studying patients with cTTP treated with Adzynma, a lab-made human ADAMTS13 protein, to better understand its safety and risks. This observational study collects and reviews medical records retrospectively for about 5 years from children and adults with cTTP who have received Adzynma treatment in routine clinical practice. The study focuses on risks of developing neutralizing antibodies against Adzynma and allergic reactions within 6 months and 7 days after initial treatment, respectively. It also aims to gather longer-term safety information and monitor pregnancy and infant outcomes in women treated with Adzynma during pregnancy. Participants medical data related to Adzynma treatment will be reviewed, including records of hypersensitivity reactions, antibody development, treatment-emergent adverse events, and pregnancy outcomes. The study evaluates these safety outcomes up to 5.5 years after treatment. No new treatments are given as part of the study. The research team monitors real-world safety data to better understand Adzynmas effects over time in patients with cTTP.

A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma