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

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.

NCT Number	NCT02675959
Lead Sponsor	New York Medical College Collaborators: University of California, Los Angeles, Medical College of Wisconsin, Johns Hopkins University, Dana-Farber Cancer Institute, Children's Hospital Los Angeles, Children's Hospital Medical Center, Cincinnati, University of Florida
Conditions	Sickle Cell Disease
Enrollment	40 participants
Start Date	2017-07-01
Primary Completion	2026-12 (estimated)
Study Completion	2027-12 (estimated)
Updated on ClinicalTrials.gov	2026-07-27