At a glance
ClinicalIndex Comparison Record- ✓Homozygous Hemoglobin S Disease, Hemoglobin S B0/+ thalassemia, Hemoglobin SC Disease, or Beta thalassemia intermedia/majora
- ✓Clinically significant neurologic event (stroke) or neurologic deficit >24 hours with infarct on cerebral MRI
- ✓Acute chest syndrome in preceding two years with failed, non-compliant, or declined hydroxyurea, or prior chronic RBC transfusion
- ✓Recurrent painful events (at least 3 in 2 years prior or prior to chronic transfusion)
- ✕Receiving concomitant systemic anticoagulants or fibrinolytic therapies
- ✕Previously known hypersensitivity reaction to defibrotide
- ✕Pregnant or breast-feeding
- ✕Documented uncontrolled infection at time of study entry
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Safety and Efficacy of Prophylactic Defibrotide in Children, Adolescents, and Young Adults With Sickle Cell Disease or Beta Thalassemia Following MAC and Haploidentical Stem Cell Transplantation Utilizing CD34 Enrichment and T-Cell (CD3) Addback
In Brief
A Phase 2 clinical trial evaluating Defibrotide for Sickle Cell Disease. Currently recruiting, targeting 40 participants across 4 sites.
Detailed 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.
Study Details
Timeline
Interventions
defibrotide will be given prophylactically prior to AlloSCT to determine if it decreases the incidence of SOS in this high risk population, and determine that it is safe and feasible to give along with myeloimmunoablative therapy and allogeneic transplant.