Defibrotide Dose-escalation for SOS Post-HSCT

This study is testing if increasing the dose of defibrotide is safe and tolerable for patients who develop sinusoidal obstructive syndrome (SOS) or veno-occlusive disease (VOD) after a stem cell transplant (HCT). This condition affects the liver and can also impact the kidneys or lungs. The study is for patients whose SOS/VOD has not improved or has worsened with standard doses of defibrotide. Researchers want to see how many serious side effects related to defibrotide occur within 100 days. The study aims to enroll 20 participants between 1 month and 75 years old, of all genders, who meet specific criteria for SOS/VOD and have not responded to standard defibrotide treatment.

Study design: This is an interventional study with a planned enrollment of 20 participants. It is designed to evaluate the safety and tolerability of increasing doses of defibrotide within individual patients.
What's involved: Not specified in the trial record.
Compensation: Not stated in the trial record.
Follow-up: Participants will be followed to measure adverse events related to defibrotide at 100 days.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

Defibrotide Dose-escalation for SOS Post-HSCT

Recruiting

PHASE2Ages 1–75InterventionalTreatment

New York Medical College

~20 participants

Updated 2026-04-15 on ClinicalTrials.gov

What's tested:Defibrotide

At a glance

Recruiting sites

1 of 1 listed site is recruiting right now

RecruitingSuspended, closed, or not yet open

What they're measuring

To determine the incidence of grade 3 or 4 adverse events related to defibrotide

Measured over 100 days

Conditions

Sinusoidal Obstruction Syndrome

Veno-occlusive Disease

Where it's being run

1 sites across 1 states

New York1

Study leadership

Mitchell Cairo, MD · PRINCIPAL_INVESTIGATOR · New York Medical College

Who to contact

Mitchell Cairo, MD

[email protected]

914-594-2150

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Eligibility criteria

Inclusion

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

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.

What this trial measures

To determine the incidence of grade 3 or 4 adverse events related to defibrotide100 days
grade 3 and 4 adverse events possible or probably related to defibrotide will be collected

Trialytics