Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
Status:
Recruiting
Trial end date:
2024-12-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
New York Medical College
Collaborators:
Baylor College of Medicine Children's Hospital Los Angeles Dana-Farber Cancer Institute Johns Hopkins University Medical College of Wisconsin Tufts Medical Center University of California, Los Angeles University of Michigan