Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy for Neuroblastoma
Not yet recruiting
Jazz Pharmaceuticals
Phase 2
2024-06-01
This phase II trial tests how well defibrotide works in preventing transplant-associated
thrombotic microangiopathy (TA-TMA) in patients with high-risk neuroblastoma undergoing
tandem transplants (hematopoietic stem cell transplant [HSCT]). TMA is a potential
life-threatening complication of stem cell transplant. TMA is a possible side effect of the
chemotherapy (conditioning regimen) patients receive to help treat high-risk neuroblastoma,
because these medicines can sometimes damage the blood vessel walls in the body. This damage
leads to formation of tiny blood clots in organs, especially the kidney. This then causes
organ damage and leads to problems with how they function. This study may help researchers
learn how defibrotide may help prevent TMA before it starts, or help treat it once it starts
among patients with high-risk neuroblastoma undergoing tandem transplants.
Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy for Neuroblastoma
Not yet recruiting
University of California, San Francisco
Phase 2
2024-06-01
This phase II trial tests how well defibrotide works in preventing transplant-associated
thrombotic microangiopathy (TA-TMA) in patients with high-risk neuroblastoma undergoing
tandem transplants (hematopoietic stem cell transplant [HSCT]). TMA is a potential
life-threatening complication of stem cell transplant. TMA is a possible side effect of the
chemotherapy (conditioning regimen) patients receive to help treat high-risk neuroblastoma,
because these medicines can sometimes damage the blood vessel walls in the body. This damage
leads to formation of tiny blood clots in organs, especially the kidney. This then causes
organ damage and leads to problems with how they function. This study may help researchers
learn how defibrotide may help prevent TMA before it starts, or help treat it once it starts
among patients with high-risk neuroblastoma undergoing tandem transplants.
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