Overview

Defibrotide TMA Prophylaxis Pilot Trial

Status:
Completed
Trial end date:
2020-07-31
Target enrollment:
0
Participant gender:
All
Summary
Thrombotic microangiopathy (TMA) is a common complication in the stem cell transplant population. Certain populations within the hematopoietic stem cell transplant (HSCT) population are at a higher risk than others. Defibrotide is an endothelial stabilizing agent which may prevent the endothelial damage that triggers TMA in HSCT patients. The feasibility, safety, and efficacy of defibrotide prophylaxis in a pediatric transplant population is unknown. Twenty five patients age 0 to 30 years receiving autologous or allogeneic hematopoeitic stem cell transplant who meet TMA high risk criteria will be enrolled. Patients will receive Defibrotide for 28-35 days starting before conditioning, and will be closely monitored for any adverse events up through 6 months post-transplant. The feasibility of administering defibrotide will be evaluated as well as incidence of TMA.
Phase:
Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
University of California, San Francisco
Treatments:
Defibrotide
Criteria
Inclusion Criteria

1. Age 0-30 years of age

2. Life expectancy > 6 months

3. Eastern Cooperative Oncology Group or Karnofsky Performance Status >40

4. Meets minimum organ function requirements per institutional standard of care guiding
clearance for autologous or allogeneic stem cell transplantation.

5. Patients must meet TMA High-Risk criteria 5A or 5B below:

5A. Patients undergoing tandem autologous transplant with thiotepa in one or more of the
conditioning regimens

OR:

5B. . Patients with at least 3 of the following characteristics:

1. >10 years of age

2. Non-Caucasian race/ Hispanic ethnicity

3. Undergoing haploidentical transplant

4. Minor ABO blood group mismatch

Exclusion Criteria:

1. Age >30 years

2. Life expectancy < 6 months

3. Known bleeding diathesis or bleeding risk deemed by the treating physician to be a
contraindication to administration of anticoagulants.

4. Known hypersensitivity reaction to defibrotide

5. Any patient not meeting TMA High-Risk criteria

6. Pregnant women are excluded from this study because they will be receiving teratogenic
therapy as part of the stem cell transplant.