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RECRUITING
NCT05987124
PHASE2

Defibrotide Dose-escalation for SOS Post-HSCT

Sponsor: New York Medical College

View on ClinicalTrials.gov

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.

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

Key Details

Gender

All

Age Range

1 Month - 75 Years

Study Type

INTERVENTIONAL

Enrollment

20

Start Date

2024-03-20

Completion Date

2027-08-01

Last Updated

2024-03-26

Healthy Volunteers

No

Interventions

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 (1)

New York Medical College

Valhalla, New York, United States