Defibrotide Dose-escalation for SOS Post-HSCT

Description

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

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Study Overview

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Study Details

Study overview

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.

A Phase II Intrapatient Open-Label Dose Escalation Trial of Defibrotide in Hematopoietic Cell Transplantation (HCT) Recipients With Sinusoidal Obstructive Syndrome (SOS) Post-HCT Associated With Either Renal and/or Pulmonary Dysfunction With Either Refractory or Progressive Disease Following Defibrotide Therapy

Defibrotide Dose-escalation for SOS Post-HSCT

Condition
Sinusoidal Obstruction Syndrome
Intervention / Treatment

-

Contacts and Locations

Valhalla

New York Medical College, Valhalla, New York, United States, 10595

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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.

Ages Eligible for Study

1 Month to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

New York Medical College,

Mitchell Cairo, MD, PRINCIPAL_INVESTIGATOR, New York Medical College

Study Record Dates

2027-08-01