Recruiting
Phase 2

Defibrotide

Sponsor:

New York Medical College

Code:

NCT05987124

Conditions

Sinusoidal Obstruction Syndrome

Veno-occlusive Disease

Eligibility Criteria

Sex: All

Age: 0 - 70+

Healthy Volunteers: Not accepted

Interventions

Defibrotide

Study Details

Brief summary:

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.

Conditions

Sinusoidal Obstruction Syndrome

Veno-occlusive Disease

Study ID

NCT05987124

Start date

Mar 20, 2024

Status verified date

Apr, 2026

Completion date

Aug 1, 2028

Anticipated

Primary completion date

Aug 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 70+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • HCT recipients (Auto or Allograft)
  • SOS/VOD as defined by Cairo/Cooke Diagnostic criteria (1) (Table 3) with either renal and/or pulmonary dysfunction as defined by Cairo/Cooke Grading criteria (1) (Appendix I).
  • Unresponsive to standard defibrotide therapy as defined by at least one of the following:
  • Patients with SOS/VOD failing to obtain a complete response (CR) defined by Grade I or less by Cairo/Cooke Grading criteria (1) (Appendix I). This would therefore include patients with stable disease after at least 14 days of defibrotide or partial response after at least 21 days of defibrotide (25mg/kg/day).
  • Progressive disease defined by progression of at least one grade or more from diagnostic grade as defined by Cairo/Cooke Grading criteria (1) (Appendix I) following at least 7 days of defibrotide (25mg/kg/day).
  • Age 1 month - 75 years

Exclusion Criteria:

  • Patients who did not receive HCT.
  • Concomitant systemic anticoagulation (excluding central venous line management, fibrinolytic instillation for central venous line occlusion, management of intermittent dialysis or ultrafiltration of CVVH).
  • Active bleeding and/or hemorrhage of at least grade 2 and above.
  • History of development of Grade III/IV anaphylaxis probably or directly secondary to defibrotide.
  • Female patients who are pregnant or breast feeding.

Study Design

Enrollment

20 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Defibrotide

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):

Interventions

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.

Primary outcome measure

  • To determine the incidence of grade 3 or 4 adverse events related to defibrotide [ Time Frame: 100 days ]

Central Contacts and Locations

Central contacts

Mitchell Cairo, MD

914-594-2150mitchell_cairo@nymc.edu

Lauren Harrison, MSN

617-285-7844lauren_harrison@nymc.edu

Locations

New York Medical College

Recruiting

Vallhala, New York, United States, 10595

Contacts

Mitchell S Cairo, MD

914-594-2150mitchell_cairo@nymc.edu

Principal Investigator:

Mitchell S. Cairo, MD

More Information

Sponsor

New York Medical College

Last update posted

Apr 15, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by New York Medical College on 2026-04-15.