Defibrotide Dose-escalation for SOS Post-HSCT
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-75
SponsorNew York Medical College
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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).
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).
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Defibrotide
DrugHCT 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.
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: DefibrotideExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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
Time frame
100 daysLocations
1Map coordinates are unavailable for these locations. Locations are shown below instead.
New York Medical CollegeRecruiting10595, Vallhala, New York
United States
United StatesSponsors and contacts
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Source ClinicalTrials.gov