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Completed Phase 2 Interventional Results available

Defibrotide TMA Prophylaxis Pilot Trial

ClinicalTrials.gov ID: NCT03384693

Public ClinicalTrials.gov record NCT03384693. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 21, 2026, 7:53 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Pilot Trial of Using Pre-Transplant Risk Stratification and Prophylactic Defibrotide to Prevent Serious Thrombotic Microangiopathy in High-Risk Hematopoietic Stem Cell Transplant Patients

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT03384693
Recruitment status
Completed
Study type
Interventional
Phase
Phase 2
Enrollment
25 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
Up to 30 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Apr 30, 2018
Primary completion
Jul 30, 2020
Completion
Jul 30, 2020
Last update posted
Sep 15, 2021

2018 – 2020

United States locations

U.S. sites
1
U.S. states
1
U.S. cities
1
Facility City State ZIP Site status
Benioff Children's Hospital at UCSF Medical Center San Francisco California 94143

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT03384693, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Sep 15, 2021 · Synced Sep 21, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT03384693 live on ClinicalTrials.gov.

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