Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy for Neuroblastoma (NCT06182410) | Clinical Trial Compass
WithdrawnPhase 2
Defibrotide Prophylaxis of Transplant Associated-Thrombotic Microangiopathy for Neuroblastoma
Stopped: Industry sponsor decision
0Started 2024-08-01
Plain-language summary
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.
Who can participate
Age range1 Year – 18 Years
SexALL
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Inclusion criteria
✓. Age: 1 - \< 18 years old.
✓. Participants must have high-risk neuroblastoma according to Children's Oncology Group (COG) risk classification at the time of initial diagnosis. Participants who were initially considered low or intermediate risk, but then reclassified as high risk are also eligible.
✓. Participants may have had salvage therapies (i.e., meta-iodobenzylguanidine (MIBG), dinutuximab) after induction but cannot have progressive disease at start of 4.
✓. Participants may be enrolled in upfront neuroblastoma protocol, but this is not required.
✓. Upfront MIBG or other therapies is not a contraindication.
✓. Organ function per institutional standard of care (SOC) guiding clearance for autologous HSCT.
✓. Written informed consent (and assent when applicable) obtained from participant or participant's legal representative and ability for subject to comply with the requirements of the study.
Exclusion criteria
✕. Life expectancy \< 6 months.
What they're measuring
1
Cumulative incidence of transplant-associated thrombotic microangiopathy (TA-TMA)
✕. Pregnant, breastfeeding, or unwilling to practice birth control during participation in the study.
✕. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
✕. Known bleeding diathesis or bleeding risk deemed by the treating physician to be a contraindication to administration of defibrotide or on concomitant therapeutic anticoagulation. Administration of heparin and/or alteplase for central line maintenance is allowed.