Open Actively Recruiting

Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease


Brief Summary

This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD.

Primary Purpose
Study Type
Phase 2


Healthy Volunteers
Minimum Age
6 Months
Maximum Age
34 Years

Inclusion Criteria:

  • Disease: Homozygous Hemoglobin S Disease, or Hemoglobin S B0/+ thalassemia, or Hemoglobin SC Disease, or Beta thalassemia intermedia/majora
  • Patients must demonstrate one or more of the following Sickle Cell Disease Complications (or patients in Cohort 2 can meet other high risk criteria instead)
  • Clinically significant neurologic event (stroke) or any neurologic deficit lasting >24 hours that is accompanied by an infarct on cerebral MRI
  • Acute chest syndrome in the preceding two year period prior to enrollment that have failed, been non-compliant or declined hydroxyurea treatment, or prior to chronic RBC transfusion therapy, exchange transfusion or erythrocyte pheresis.
  • Recurrent painful events (at least 3 in the 2 years prior to enrollment or prior to chronic chronic RBC transfusion therapy, exchange transfusion or erythrocyte pheresis).
  • Abnormal TCD study requiring starting on chronic transfusion therapy and/or exchange transfusions.
  • At least one silent infarct lesion on a MRI scan of the head. Or (directly or probably related to SCD)
  • Sickle Cell nephropathy;
  • Splenic sequestration requiring RBC transfusion;
  • Aplastic crisis requiring RBC transfusion;
  • Avascular necrosis of the hip diagnosed by MRI;
  • Two episodes or more of leg ulcerations;
  • Recurrent priapism .
  • Infant dactylitis.
    • OR for Cohort #2 ONLY: Patient must be between 18 and 34.99 years of age, patients must demonstrate at least two of the following:
  • WBC > 13,500 cells/microliter at baseline when not acutely ill (on two separate occasions) > 2 weeks from a VOC event or hospitalization.
  • Tricuspid Regurgitant Jet Velocity (TRV) > 3.0 m/s
  • Requiring Chronic Monthly Transfusions ( > 12 transfusions in the 12 months)
  • History of sepsis
  • N-terminal pro-brain natriuretic peptide (NT-proBNP) > 160 ng/L at clinical baseline when not acutely ill or hospitalized.
  • all patients must meet disease, age, organ function and donor criteria;

Exclusion Criteria:

  • Patients who are receiving concomitant systemic anticoagulants and/or fibrinolytic therapies.
  • Patients with a previously known hypersensitivity reaction to defibrotide.
  • Females who are pregnant or breast-feeding are not eligible
  • SCD Patients with documented uncontrolled infection at the time of study entry are not eligible.
  • SCD patients who have an unaffected HLA matched family donor willing to proceed to donation will not be eligible for this study.
  • Karnofsky or Lansky (age appropriate) Performance Score <50% (hemiplegia alone secondary to a previous stroke is not an exclusion)
  • Demonstrated lack of compliance with medical care.
  • Patients with clinically significant fibrosis or cirrhosis of the liver will not be eligible.
  • Patients who have previously received a HSCT will not be eligible.
  • Patients with contraindications to the use of defibrotide

Join this Trial

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Study Stats
Protocol No.
Blood Disorders
Belen Ramirez
  • UCLA Westwood
For Providers
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