Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA
Trial Parameters
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Brief Summary
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).
Eligibility Criteria
Inclusion Criteria: 1. Age at least 28 days and less than 18 years prior to informed consent (Visit 0). 2. Have informed consent from at least one parent or legal guardian as required by local law and regulation. Patient informed consent will be required if the patient has reached the local legal age of majority. 3. Assent from patients as required by local law and regulation. 4. Have received an allogeneic hematopoietic stem cell transplant for the treatment of benign or malignant disease. 5. Have a diagnosis of HSCT-TMA defined as meeting both of the following criteria: * Platelet count \< 50,000/mL or a decrease in platelet count \> 50% from the highest value obtained following transplant. * Evidence of microangiopathic hemolysis (presence of schistocytes, serum lactate dehydrogenase \[LDH\] \> upper limit of normal (\[ULN\], or haptoglobin \< lower limit of normal \[LLN\]) 6. Have at least one of the following HSCT-TMA high-risk criteria: * HSCT-TMA persistence \> 2 weeks following