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Defibrotide for Sickle Cell Disease Patients Undergoing Stem Cell Transplant

This study is looking at whether a medicine called defibrotide can help prevent a serious liver complication called sinusoidal obstructive syndrome (SOS) in children and young adults with sickle cell disease or beta thalassemia. These patients are undergoing a stem cell transplant, which can sometimes lead to SOS. Defibrotide has been shown to help treat SOS, and this study wants to see if giving it beforehand can prevent it. The study will monitor for any side effects of defibrotide and track whether patients develop SOS for up to one year. You might be able to join if you are between 6 months and 21 years old, have certain types of sickle cell disease or beta thalassemia, and have experienced specific complications like a stroke.

Study design
This study plans to enroll 40 participants. It is an interventional study, meaning participants will receive a specific treatment.
What's involved
You will have daily physical exams and laboratory tests while in the hospital, and then as needed. You will be monitored for side effects and SOS for up to one year.
Compensation
Not stated in the trial record.
Follow-up
All patients will be monitored for side effects for 100 days and for the development of SOS for 1 year.

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NCT02675959

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

Recruiting
PHASE2Ages 6–21InterventionalTreatment
New York Medical College
~40 participants
Updated 2026-07-27 on ClinicalTrials.gov
What's tested:Defibrotide

At a glance

Recruiting sites
4 of 4 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
All patients will be monitored for known and unknown side effects of defibrotide with daily physical exams while in the hospital and then as needed in addition to daily laboratory values including chemistries, hematology labs as needed
Measured over 100 days
+1 more outcome measured

Conditions

Sickle Cell Disease

Explore related trials

Beta ThalassemiaSickle Cell DiseaseBeta ThalassemiaHemoglobin S

Where it's being run

4 sites across 4 states
California1
Florida1
New York1
Wisconsin1

Study leadership

  • Mitchell Cairo, MD · PRINCIPAL_INVESTIGATOR · New York Medical College

Who to contact

Mitchell S Cairo, MD
[email protected]
914-594-2150
Email the study team

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Eligibility criteria

Inclusion

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
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 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.
all patients must meet disease, age, organ function and donor criteria;

Exclusion

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
Patients with documented uncontrolled infection at the time of study entry are not eligible.
Patients who have an unaffected HLA matched sibling 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

What this trial measures

  • All patients will be monitored for known and unknown side effects of defibrotide with daily physical exams while in the hospital and then as needed in addition to daily laboratory values including chemistries, hematology labs as needed100 days

    Patients will be given Defibrotide prophylaxis starting 10 days before the stem cell infusion at 6.25 mg/kg IV q6h and continue through Day +21.

  • All patients will be monitored for the development of SOS.1 year

    All patients will get daily lab values while in patients and then as needed to monitor for elevation in liver function tests and other abnormal chemistry or hematology values. Imaging on the liver will be performed as needed to determine if they develop SOS with defibrotide.