Clinical Trials Logo

Clinical Trial 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.


Clinical Trial Description

Sinusoidal obstructive syndrome (SOS) has a reported mean incidence of 13.7% and even among those undergoing reduced intensity conditioning regimens is approximately 9%. SOS is a clinical syndrome characterized by painful hepatomegaly, jaundice, ascites, fluid retention, and weight gain. The onset is usually before day 35 after stem cell infusion. SOS ranges in severity from a mild reversible disease to a severe syndrome associated with multiorgan failure (MOF) and death. Prior to the introduction of defibrotide, severe SOS was nearly universally fatal with a mortality rate approaching 100% by day 100 after allo-SCT. The diagnosis of SOS/VOD is clinical and should be considered in any patient who has undergone hematopoietic stem cell transplantation and develops liver dysfunction. Patients with mild or moderate disease have reasonably good outcomes with supportive therapy alone while in contrast prognosis is much worse in severe SOS which occurs in about 25-30% cases. Defibrotide is the only established Food and Drug Administration (FDA) approved therapy to treat SOS. It is now approved for use in adults and children with SOS with renal or pulmonary dysfunction following HCT. The standard treatment is 25 mg/kg/day in 4 divided doses of 6.25 mg/kg for 21 days. However, responses are frequently brisk and complete in many patients thus it has been postulated that in responding patients this treatment course could be abbreviated given the risk for adverse events such as hypotension/shock and hemorrhage. This is an open label pilot study evaluating an abbreviated 5 (as compared to 21) day course of defibrotide in patients with confirmed SOS. The primary outcome is day 100 survival as compared to historical data. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04313036
Study type Interventional
Source Loyola University
Contact Patrick A Hagen, MD, MPH
Phone 708-327-3157
Email patrick.hagen@lumc.edu
Status Recruiting
Phase Phase 2
Start date March 11, 2021
Completion date August 1, 2022

See also
  Status Clinical Trial Phase
Recruiting NCT05611307 - Late Subclinical Cardiovascular Disease in Testicular Cancer Survivors
Recruiting NCT03818334 - Post Transplant Cyclophosphamide in Matched Unrelated Donor Stem Cell Transplantation for Hematological Malignancies Phase 2/Phase 3
Withdrawn NCT05895201 - High-Dose Post-Transplant Cyclophosphamide, Bortezomib, and Sitagliptin for the Prevention of GVHD Phase 1/Phase 2
Completed NCT04687982 - Feasibility and Efficacy of Modified Donor Lymphocytes Infusion (CD45RA Negative Selected) After Haploidentical Transplantation With Post-transplantation Cyclophosphamide in Patients With Hematological Malignancies (ONC-2016-002). N/A
Recruiting NCT06416761 - Genetics in the Progression of Nephropathies
Completed NCT03858530 - Ultrasound Elastography to Predict Development of SOS Phase 4
Completed NCT03200626 - Observational Study to Evaluate Hematopoietic Progenitor Cell Mobilization in Patients With Newly Diagnosed Multiple Myeloma
Recruiting NCT05709912 - Mobile CARE-App to Promote Coping for Caregivers of Patients Receiving Stem Cell Transplant N/A
Completed NCT05175222 - Assessment of Efficacy of LightWalker (Nd:Yag, Er:Yag) Laser Therapy on Incidence, Severity and Duration of Oral Mucositis in Patients Undergoing Hematopoietic Stem Cell Transplantation (HSCT). Prospective Randomized Open-label Study N/A
Recruiting NCT05316740 - Vaginal Microbiome and the Development of Vulvovaginal Graft Versus Host Disease
Recruiting NCT05629676 - Virtual Reality Intervention for Patients Undergoing BMT N/A
Recruiting NCT05694910 - Reiki Effects on Bone Marrow Transplant Patients N/A
Completed NCT05352789 - Nutrition and Metabolic Prehabilitation in HSCT Patients UK and ROI. BSBMT Multi-centre Survey.
Withdrawn NCT03963999 - Validating Ultrasound Biomarkers for Hepatic Sinusoidal Obstruction Syndrome in Pediatric Hematopoietic Cell Transplant Patients Phase 4
Recruiting NCT03865589 - Using Ultrasound Elastography to Predict Development of Hepatic Sinusoidal Obstruction Syndrome N/A