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RecruitingPhase 2

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

The Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents, and Young Adults With Sickle Cell Disease or Beta Thalassemia Following Myeloablative Conditioning (MAC) and Haploidentical or Matched Unrelated Donor (MUD) Stem Cell Transplantation Utilizing CD34 Enrichment and T-Cell (CD3) Addback

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
6 Months to 21 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether defibrotide is safe and helps prevent blocked small blood vessels in the liver during a donor stem cell transplant. It is for high-risk patients with sickle cell disease or beta thalassemia, an inherited blood disorder. Donors are partly matched family members or matched unrelated people.

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 or MUD 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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Have one of these blood disorders: hemoglobin SS, S beta-zero or beta-plus thalassemia, hemoglobin SC, or beta thalassemia intermedia or major.
  • Meet the study’s age, organ health, and donor rules. Patients must also have at least one of the listed sickle cell complications.
  • Have had a stroke, or nerve or brain problems lasting over 24 hours with brain damage on MRI; a silent stroke seen on MRI also counts.
  • Have had acute chest syndrome, a serious lung problem, in the past 2 years with the listed hydroxyurea treatment conditions, or before starting regular blood transfusions or blood exchanges.
  • Have had at least 3 pain attacks in the past 2 years or before regular transfusions or blood exchanges; or an abnormal brain blood-flow scan requiring these treatments.
  • Have sickle cell kidney damage, blood trapped in the spleen requiring a transfusion, or a pause in red blood cell production requiring a transfusion.
  • Have hip bone damage from poor blood flow confirmed by MRI, or at least 2 episodes of leg sores.
  • Have repeated long-lasting erections or painful swelling of fingers or toes in infancy.

Who can't join

  • Take medicines that thin the blood or dissolve blood clots throughout the body.
  • Have a known allergy to defibrotide or another medical reason that makes it unsafe to use.
  • Are pregnant or breastfeeding.
  • Have an uncontrolled infection when joining the study.
  • Have a sibling without the disease who is a full tissue match and is willing to donate.
  • Have a daily activity score below 50%. One-sided paralysis from a past stroke alone does not rule you out.
  • Have a history of not following medical care.
  • Have serious liver scarring or have already had a blood stem cell transplant.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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

Study sites (4)

CA

FL

NY

WI