Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant

Inclusion Criteria:

1. 12 years of age or older at time of consent/assent.
2. Received HSCT within the past 12 months
3. Diagnosis of TMA that persists for at least 72 hours despite initial management
4. A TMA diagnosis based on meeting the select criteria during the Screening Period and/or <=14 days prior to the Screening Period.
5. Body weight ≥ 30 kilograms at Screening.
6. Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.
7. Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis.
8. Participants or their legally authorized representative must be capable of giving signed informed consent or assent

Exclusion Criteria:

1. Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency
2. Shiga toxin producing Escherichia coli infection
3. Positive direct Coombs test.
4. Clinical diagnosis of disseminated intravascular coagulation (DIC).
5. Known bone marrow/graft failure.
6. Diagnosis of veno-occlusive disease.
7. Human immunodeficiency virus (HIV) infection.
8. Unresolved meningococcal disease.
9. Presence of sepsis requiring vasopressor support.
10. Pregnancy or breastfeeding.
11. Previously or currently treated with a complement inhibitor.
12. Respiratory failure requiring mechanical ventilation.
13. Acute and/or chronic heart failure.
14. Participation in an interventional treatment study of any therapy for TMA.