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Defibrotide TMA Prophylaxis Pilot Trial

NCT03384693 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 25

Last updated 2021-09-16

Study results available
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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.

Conditions

  • Thrombotic Microangiopathies

Interventions

DRUG

Defibrotide

Defibrotide is an anticoagulant and fibrinolytic agent that has been shown to be an effective treatment in other endothelial disorders such as hepatic veno-occlusive disease.

Sponsors & Collaborators

Principal Investigators

  • Christine Higham, MD · University of California, San Francisco

Study Design

Allocation
NA
Purpose
PREVENTION
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-05-01
Primary Completion
2020-07-31
Completion
2020-07-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03384693 on ClinicalTrials.gov