Sinusoidal Obstruction Syndrome for Stem Cell Transplant Patients Biomarker Study

Completed

• Conditions: Sinusoidal Obstruction Syndrome
• Interventions: Other: Serial Blood Draws

• Registration Number: NCT03132337
• Lead Sponsor: Indiana University

Brief Summary

The goal of this is to learn more about stem cell transplant and complications that some people have after their transplants, in particular sinusoidal obstruction syndrome (SOS), also called veno-occlusive disease of the liver.

Detailed Description

This is a multicenter, prospective, observational trial. We will measure biomarkers and determine thresholds that will predict increased risk for SOS in pediatric patients receiving HCT or high intensity chemotherapy/irradiation with the future goal of a randomized, interventional, open-label, multicenter trial that will test the preemptive use of defibrotide for prevention of SOS in an enriched high-risk population.

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2017-04-01
• Study First Submitted: 2017-04-24
• Study First Submitted QC: 2017-04-26
• Study First Posted: 2017-04-27
• Primary Completion: 2021-01-14
• Study Completion: 2021-01-14
• Status Verified: 2023-12
• Last Update Submitted: 2023-12-18
• Last Update Posted: 2023-12-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 80

Inclusion Criteria

Age ≤ 25 years undergoing HCT for any reason who fulfill any ONE (1) of the following criteria:

1. History of hepatic disease as defined by:

   1. Viral hepatitis (i.e., hepatitis C virus [HCV])
   2. Liver tumor before HCT
   3. Hepatic fibrosis or cirrhosis before HCT as proven by liver biopsy
   4. High aspartate aminotransferase (AST) (> 2x ULN) before HCT (pre-transplant evaluation)
   5. High alanine transaminase (ALT) (> 2x ULN) before HCT
   6. High bilirubin (> 1.2x ULN) before HCT

2. HCT high-risk features including:

   a. Conditioning with high-risk modalities including: i. Busulfan (BU)-containing regimen particularly with oral BU + cyclophosphamide ii. TBI-containing regimen, particularly cyclophosphamide + total-body irradiation (TBI) b. ≥ 2 HCT c. Allo-HCT for leukemia > or = second relapse d. Unrelated donor (URD) HCT e. Human leukocyte antigen (HLA) mismatch HCT (less than 10 of 10 for bone marrow/peripheral blood stem cell [BM/PBSC] or anything less than 6 of 6 for UCB) f. Use of sirolimus + tacrolimus prophylaxis for GVHD

3. High-risk disease states including:

   1. Juvenile myelo-monocytic chronic leukemia (JMML)
   2. Primary hemophagocytic lymphohistiocytosis (HLH)
   3. Adrenoleukodystrophy
   4. Osteopetrosis

4. Other high-risk features including:

   1. Prior treatment with gemtuzumab ozogamicin
   2. Use of hepatotoxic drugs 1 month before HCT and during HCT
   3. Iron overload (i.e., thalassemia/sickle cell) with serum ferritin > 1000ng/ml
   4. Deficit of ATIII, T-PA (i.e., < 30% normal values), and resistance to activated protein C if clinical indication (these values do not have to be specifically checked if no clinical history)
   5. Young age < 2 years but more than 1 month

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Exclusion Criteria

Patients who are transplanted but do not fulfill any of the above mentioned criteria.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Stem Cell Transplant	Serial Blood Draws	Serial Blood Draws

Primary Outcome Measures

Name	Time	Method
SOS proteomic markers	Until the end of the study evaluation, day 180	Measure for 3 SOS proteomic markers, L-Ficolin, HA, and ST2, as early predictors of SOS incidence through study completion.

Trial Locations

Locations (4)

Indiana University; 🇺🇸 Indianapolis, Indiana, United States

Baylor College of Medicine; 🇺🇸 Houston, Texas, United States

Children's National Medical Center; 🇺🇸 Washington, District of Columbia, United States

University of Michigan; 🇺🇸 Ann Arbor, Michigan, United States