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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05987124
Other study ID # NYMC 600
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
Start date March 20, 2024
Est. completion date August 1, 2027

Study information

Verified date March 2024
Source New York Medical College
Contact Mitchell Cairo, MD
Phone 914-594-2150
Email mitchell_cairo@nymc.edu
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Recruiting
Enrollment 20
Est. completion date August 1, 2027
Est. primary completion date August 1, 2026
Accepts healthy volunteers No
Gender All
Age group 1 Month to 75 Years
Eligibility 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


Intervention

Drug:
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.

Locations

Country Name City State
United States New York Medical College Valhalla New York

Sponsors (1)

Lead Sponsor Collaborator
New York Medical College

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary To determine the incidence of grade 3 or 4 adverse events related to defibrotide grade 3 and 4 adverse events possible or probably related to defibrotide will be collected 100 days
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