Using Defibrotide to Improve Stem Cell Transplant Outcomes for Sickle Cell Disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the safety and efficacy of Defibrotide as a preventive treatment for patients with sickle cell disease undergoing haploidentical stem cell transplantation. The study focuses on patients at high risk for developing sinusoidal obstructive syndrome (SOS), a serious complication associated with this procedure. By administering Defibrotide prophylactically, the trial aims to reduce the incidence of SOS and improve overall transplant outcomes. This is a follow-up to a previous trial that laid the groundwork for this investigation.

Why it matters

Who can join: the full rules

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
* 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 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
* 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

Where it's running

• University of California Los Angeles
  Los Angeles, California, United States Recruiting
• University of Florida
  Gainesville, Florida, United States Recruiting
• New York Medical College
  Valhalla, New York, United States Recruiting
• Medical College of Wisconsin
  Milwaukee, Wisconsin, United States Recruiting

Study contacts

• Principal investigator: Mitchell Cairo, MD — New York Medical College
• Study coordinator: Mitchell S Cairo, MD
• Email: Mitchell_Cairo@nymc.edu
• Phone: 914-594-2150

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.