Defibrotide TMA Prophylaxis Pilot Trial

Completed · Phase 2

Conditions studied: Thrombotic Microangiopathies

In brief

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.

Key facts

Study ID

NCT03384693

Run by

University of California, San Francisco

People needed

25

Starts

2018-05-01

Expected to finish

2020-07-31

Last updated by the study team

2021-09-16

Who can join

Age: any, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age 0-30 years of age
• Life expectancy > 6 months
• Eastern Cooperative Oncology Group or Karnofsky Performance Status >40
• Meets minimum organ function requirements per institutional standard of care guiding clearance for autologous or allogeneic stem cell transplantation.
• 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:
• >10 years of age
• Non-Caucasian race/ Hispanic ethnicity
• Undergoing haploidentical transplant
• Minor ABO blood group mismatch

You may not qualify if…

• Age >30 years
• Life expectancy < 6 months
• Known bleeding diathesis or bleeding risk deemed by the treating physician to be a contraindication to administration of anticoagulants.
• Known hypersensitivity reaction to defibrotide
• Any patient not meeting TMA High-Risk criteria
• Pregnant women are excluded from this study because they will be receiving teratogenic therapy as part of the stem cell transplant.

Where it is running

• Benioff Children's Hospital at UCSF Medical Center — San Francisco, California, United States

Full record on ClinicalTrials.gov

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