RECRUITING

Defibrotide Dose-escalation for SOS Post-HSCT

Conditions

Sinusoidal Obstruction Syndrome Veno-occlusive Disease

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2024-03-20

Study Completion:2027-08-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05987124

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.

Ages Eligible for Study:1 Month to 75 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.

Inclusion Criteria

Exclusion 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.

Contacts and Locations

Study Contact

Mitchell Cairo, MD

CONTACT

914-594-2150

mitchell_cairo@nymc.edu

Lauren Harrison, MSN

CONTACT

617-285-7844

lauren_harrison@nymc.edu

Principal Investigator

Mitchell Cairo, MD

PRINCIPAL_INVESTIGATOR

New York Medical College

Study Locations (Sites)

New York Medical College

Valhalla, New York, 10595

United States

Collaborators and Investigators

Sponsor: New York Medical College

Mitchell Cairo, MD, PRINCIPAL_INVESTIGATOR, New York Medical College

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-03-20

Study Completion Date2027-08-01

Study Record Updates

Study Start Date2024-03-20

Study Completion Date2027-08-01

Terms related to this study

Additional Relevant MeSH Terms

Sinusoidal Obstruction Syndrome
Veno-occlusive Disease