Increasing doses of defibrotide for treating sinusoidal obstruction syndrome after transplant

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial aims to evaluate the safety and tolerability of escalating doses of defibrotide in patients suffering from sinusoidal obstruction syndrome (SOS) or veno-occlusive disease (VOD) following hematopoietic cell transplantation (HCT). The study focuses on patients who have not achieved a complete response or have shown disease progression with standard defibrotide doses. Participants will receive higher doses of the medication to assess its effectiveness in improving their condition. The trial is designed to provide insights into alternative dosing strategies for patients with severe symptoms.

Why it matters

Who can join: the full rules

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.

Where it's running

• New York Medical College
  Vallhala, New York, United States Recruiting

Study contacts

• Principal investigator: Mitchell Cairo, MD — New York Medical College
• Study coordinator: Mitchell Cairo, MD
• Email: mitchell_cairo@nymc.edu
• Phone: 914-594-2150

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.