Defibrotide Dose-escalation for SOS Post-HSCT

Part of paid clinical trials in Vallhala, New York.

Sponsor
New York Medical College
Study ID
NCT05987124
Phase
PHASE2
Status
Recruiting
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Conditions

  • Sinusoidal Obstruction Syndrome
  • Veno-occlusive Disease

Eligibility Criteria

Sex
ALL
Age
1 Month - 75 Years
Healthy Volunteers
Not accepted

Interventions

  • Defibrotide — DRUG
    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.

Study Details

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.

Key Dates

Start date
Mar 20, 2024
Status verified
Apr 2026
Primary completion
Aug 1, 2027
Completion
Aug 1, 2028

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Defibrotide
    7.1.1 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 (see 7.2 for definition of response) (Maximum of 4 dose levels) (7.1.2):

Primary Outcome Measure

To determine the incidence of grade 3 or 4 adverse events related to defibrotide [ Time Frame: 100 days ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
New York Medical CollegeVallhalaNew York10595
Mitchell S Cairo, MD
[email protected]
914-594-2150
Mitchell S. Cairo, MD (PRINCIPAL_INVESTIGATOR)

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New York Medical College· Vallhala, NY

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