NCT04313036

Brief Summary

Sinusoidal Obstruction Syndrome (SOS), also referred to as hepatic veno-occlusive disease (VOD), is rare but serious complication of allogeneic stem cell transplantation (allo-SCT). Defibrotide is the only FDA approved therapy to treat SOS and has significantly improved outcomes. When applied early, SOS symptoms often quickly improve and an abbreviated course can be applied. This study is looking at an abbreviated 5 day course of defibrotide in those patients with a complete response to therapy with the primary outcome being day 100 overall survival as compared to history data.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
24

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Mar 2021

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 16, 2020

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 18, 2020

Completed
12 months until next milestone

Study Start

First participant enrolled

March 11, 2021

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2022

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2022

Completed
Last Updated

April 23, 2021

Status Verified

April 1, 2021

Enrollment Period

1.1 years

First QC Date

March 16, 2020

Last Update Submit

April 22, 2021

Conditions

Sinusoidal Obstruction SyndromeVeno-occlusive DiseaseBone Marrow Transplant Complications

Keywords

defibrotideSinusoidalVeno-occlusiveBone Marrow Transplantallogeneic stem cell transplant

Outcome Measures

Primary Outcomes (1)

  • Day 100 overall survival

    Assess day 100 survival of 5-day defibrotide treatment for hepatic SOS/VOD in allogeneic stem cell transplant patients compared to standard 21-day treatment in the reported literature.

    100 days

Secondary Outcomes (3)

  • Complete response day 100

    100 days

  • Complete response day 5

    5 days

  • Complete response day 30

    30 days

Study Arms (1)

Defibrotide

EXPERIMENTAL

5 day course of defibrotide at standard dosing 25 mg/kg/day in 4 divided doses of 6.25 mg/kg. If not in CR by day 5, will be given for \>/= 21 days or per discretion of enrolling physician.

Drug: Defibrotide

Interventions

DefibrotideDRUG

25 mg/kg/day at 4 divided doses of 6.25 mg/kg, 2-h infusion given for 5 days, if not in CR treatment continued for \>/= 21 days or per discretion of enrolling physician.

Defibrotide

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients who underwent allogeneic stem cell transplantation
  • Age \>/= 18 years
  • Diagnosed hepatic SOS/VOD either by Baltimore Criteria or Modified Seattle Criteria including up to 60 days post-transplantation.

You may not qualify if:

  • Significant uncontrolled bleeding
  • Prior or concurrent systemic t-PA
  • Concomitant use of therapeutic heparin or other anticoagulants (except use of heparin for central access patency)
  • Hemodynamic instability (\>1 pressor gent to maintain blood pressure)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Loyola University Medical Center

Maywood, Illinois, 60153, United States

RECRUITING

MeSH Terms

Conditions

Hepatic Veno-Occlusive Disease

Interventions

defibrotide

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesVascular DiseasesCardiovascular Diseases

Study Officials

  • Patrick A Hagen, MD

    Loyola University

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Patrick A Hagen, MD, MPH

CONTACT

708-327-3157patrick.hagen@lumc.edu

Mary Lee

CONTACT

708-327-2241mlee@luc.edu

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: The goal of this study is to assess the 100-day survival of a 5-day defibrotide treatment for hepatic SOS in allogeneic stem cell transplant patients compared to the standard 21-day treatment. It is hypothesized that the 5-day treatment will have non-inferior 100-day survival rates to the 21-day treatment. Historically, 100-day survival has been 58.9% in moderate and 38.2% in severe SOS. Investigators believe that the null survival for the 21-day defibrotide treatment is 50%. Investigators wish to determine non-inferiority of the 5-day treatment with a 5% margin of error.
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Assistant Professor

Study Record Dates

First Submitted

March 16, 2020

First Posted

March 18, 2020

Study Start

March 11, 2021

Primary Completion

May 1, 2022

Study Completion

August 1, 2022

Last Updated

April 23, 2021

Record last verified: 2021-04

Data Sharing

IPD Sharing
Will not share

Locations

US(1)