Actively Recruiting
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
Led by New York Medical College · Updated on 2025-05-23
40
Participants Needed
4
Research Sites
543 weeks
Total Duration
On this page
Sponsors
N
New York Medical College
Lead Sponsor
U
University of California, Los Angeles
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of homozygous hemoglobin S disease, hemoglobin S B0/+ thalassemia, hemoglobin SC disease, or beta thalassemia intermedia/major
- Patients must have one or more sickle cell disease complications such as stroke with MRI infarct, acute chest syndrome in the past 2 years with failed or declined treatments, recurrent painful events (3 or more in 2 years), abnormal TCD requiring transfusions, silent infarct on MRI, sickle cell nephropathy, splenic sequestration needing transfusion, aplastic crisis needing transfusion, avascular necrosis diagnosed by MRI, two or more leg ulcer episodes, recurrent priapism, or infant dactylitis
- For Cohort 2 only: patients aged 18 to 34.99 with at least two of the following: WBC >13,500 on two occasions when not ill, tricuspid regurgitant jet velocity >3.0 m/s, chronic monthly transfusions (>12 in 12 months), history of sepsis, or NT-proBNP >160 ng/L at baseline when not ill
- All patients must meet disease, age, organ function, and donor criteria
You will not qualify if you...
- Receiving systemic anticoagulants or fibrinolytic therapies
- Known hypersensitivity to defibrotide
- Females who are pregnant or breastfeeding
- Uncontrolled infection at study entry
- Having an unaffected HLA-matched family donor willing to donate
- Karnofsky or Lansky performance score below 50% (except hemiplegia from prior stroke)
- Demonstrated lack of compliance with medical care
- Clinically significant liver fibrosis or cirrhosis
- Previous hematopoietic stem cell transplant
- Contraindications to defibrotide use
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
University of California Los Angeles
Los Angeles, California, United States, 90095
Actively Recruiting
2
University of Florida
Gainesville, Florida, United States, 32610-0278
Actively Recruiting
3
New York Medical College
Valhalla, New York, United States, 10595
Actively Recruiting
4
Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
Research Team
M
Mitchell S Cairo, MD
CONTACT
E
Erin Morris, RN
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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