Recruiting
Phase 2

Defibrotide

Sponsor:

Loyola University

Code:

NCT04313036

Conditions

Sinusoidal Obstruction Syndrome

Veno-occlusive Disease

Bone Marrow Transplant Complications

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Defibrotide

Study Details

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.

Conditions

Sinusoidal Obstruction Syndrome

Veno-occlusive Disease

Bone Marrow Transplant Complications

Study ID

NCT04313036

Start date

Mar 11, 2021

Status verified date

Apr, 2021

Completion date

Aug 1, 2022

Anticipated

Primary completion date

May 1, 2022

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Design

Enrollment

24 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Defibrotide

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.

Interventions

Defibrotide

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.

Primary outcome measure

  • Day 100 overall survival [ Time Frame: 100 days ]

Central Contacts and Locations

Central contacts

Patrick A Hagen, MD, MPH

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

Mary Lee

708-327-2241mlee@luc.edu

Locations

Loyola University Medical Center

Recruiting

Maywood, Illinois, United States, 60153

Contacts

Patrick A Hagen, MD

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

Mary Lee, RN

708-327-2241mlee@luc.edu

Principal Investigator:

Patrick Hagen, MD

More Information

Sponsor

Loyola University

Last update posted

Apr 23, 2021

Last verified

Apr, 2021

Keywords

  • defibrotide
  • Sinusoidal
  • Veno-occlusive
  • Bone Marrow Transplant
  • allogeneic stem cell transplant

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Loyola University on 2021-04-23.