Who can enter
- Children with high-risk hematopoietic stem cell transplant associated thrombotic microangiopathy (HSCT-TMA)
- Age: 28 days to 18 years old
Goal
The goal of this study is to look at how safe the new study drug narsoplimab is for the treatment of children with hematopoietic stem cell transplant associated thrombotic microangiopathy (HSCT-TMA). We will also look at how well it works.
Background
This study will use an investigational study drug called narsoplimab in children with a condition called thrombotic microangiopathy (TMA). TMA is a rare blood-related condition that results in abnormal blood clots (clumps that occur when blood hardens from a liquid to a solid) in small blood vessels of the body.
There are several types of TMA. The type that will be part of this study is hematopoietic stem cell transplant associated TMA (HSCT-TMA). This is a form of TMA that sometimes occurs in patients who have received an allogeneic hematopoietic stem cell transplant. This type of transplant is a procedure in which a person receives stem cells (cells from which all blood cells develop) from a genetically similar, but not identical, donor.
HSCT-TMA is a serious multisystem disease (various organs may be affected) influencing post-transplant outcomes. Mortality can be 90% or higher in severe high-risk cases. No treatment is approved for HSCT-TMA, and this condition represents a serious unmet medical need. Modification of the increased activity of the immune system is generally performed, and patients may improve with this conservative treatment alone.
The purpose of this study is to test the safety and describe the effect of narsoplimab, an ‘investigational drug’, in patients with this type of disease. ‘Investigational drug’ means that the drug has not been approved by the US Food and Drug Administration (FDA).