A Phase 2 interventional study of Defibrotide in Sinusoidal Obstruction Syndrome and Veno-occlusive Disease, sponsored by New York Medical College. Recruiting at 1 site in United States. Open to participants aged 1 Month to 75 Years. Per ClinicalTrials.gov, last updated 2026-04-15.
Sponsored by New York Medical College · Phase 2, Interventional, and Treatment
This research study is being done to determine the safety and tolerability of increasing doses of defibrotide within a single patient with sinusoidal obstructive syndrome (SOS)/veno-occlusive disease (VOD) after hematopoietic cell transplantation (HCT) associated with either kidney and/or lung impairment that has not obtained a complete response (CR) or progressed in severity with standard doses of defibrotide.
23 studies on the registry are indexed under Hepatic Veno-Occlusive Disease; 3 are open to participants now.
This study's planned enrollment of 20 is below the median of 80 across 15 interventional studies indexed under Hepatic Veno-Occlusive Disease.
Browse Hepatic Veno-Occlusive Disease studies →New York Medical College is the lead sponsor of 69 studies on the registry; 23 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 0 (0%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
7.1.1 HCT recipients with SOS/VOD and renal and/or pulmonary dysfunction with either PR after 21 days of standard doses of defibrotide (25mg/kg/day) or SD, after 14 days of standard doses of defibrotide (25mg/kg/day) progressive disease after 7 days on defibrotide (25mg/kg/day) will undergo intra-patient dose escalation every 4 days until a complete response is obtained up until the highest dose level of 100mg/kg/day at which point an endpoint of CR, PR or SD will be sought (see 7.2 for definition of response) (Maximum of 4 dose levels) (7.1.2):
Drug: Defibrotide
HCT recipients with SOS/VOD and renal and/or pulmonary dysfunction with either PR after 21 days of standard doses of defibrotide (25mg/kg/day) or SD, after 14 days of standard doses of defibrotide (25mg/kg/day) progressive disease after 7 days on defibrotide (25mg/kg/day) will undergo intra-patient dose escalation every 4 days until a complete response is obtained up until the highest dose level of 100mg/kg/day at which point an endpoint of CR, PR or SD will be sought(Maximum of 4 dose levels). Defibrotide will be administered in D5W or 0.9% NaCl via IV infusion over 2 hours q6 hours.
Also known as: Defitelio
To determine the incidence of grade 3 or 4 adverse events related to defibrotide
grade 3 and 4 adverse events possible or probably related to defibrotide will be collected
Time frame: 100 days
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Hepatic Veno-Occlusive Disease→
New York Medical College