This is a Phase 2, uncontrolled, single-dosing regimen study in pediatric patients from 28 days to less than 18 years of age with high risk HSCT-TMA. At least 4 patients will be required from each of 3 age cohorts:~28 days to <2 years of age, 2 years to <12 years of age, and 12 years to <18 years of age.~Treatment will be for 8 weeks and patients will be followed for up to 52 weeks.
The purpose of this study is to evaluate the safety and efficacy of narsoplimab in pediatric patients with thrombotic microangiopathies (TMA) following hematopoietic stem cell transplant (HSCT).