Sinusoidal Obstruction Syndrome for Stem Cell Transplant Patients Biomarker Study

Sinusoidal Obstruction Syndrome Clinical Trial

— SOSBiomarker  

Official title:

Biomarkers of Endothelial Dysfunction in Pediatric Patients Receiving High Intensity Chemotherapy/Irradiation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03132337
• Other study ID #: 1701020549
• Status: Completed
• Start date: April 1, 2017
• Est. completion date: January 14, 2021

Study information

• Verified date: December 2023
• Source: Indiana University
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 80
• Est. completion date: January 14, 2021
• Est. primary completion date: January 14, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 25 Years

Eligibility

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

Exclusion Criteria:

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

Related Conditions & MeSH terms

• Hepatic Veno-Occlusive Disease
• Sinusoidal Obstruction Syndrome
• Syndrome

Intervention

Other:
• Serial Blood Draws
Day 0 and Day 3 blood draw for SOS biomarkers. If subject develops SOS blood draw prior to dose of Defibrotide, Day 14 after Defibrotide and Day 21 after Defibrotide.

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan
United States	Baylor College of Medicine	Houston	Texas
United States	Indiana University	Indianapolis	Indiana
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Indiana University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	SOS proteomic markers	Measure for 3 SOS proteomic markers, L-Ficolin, HA, and ST2, as early predictors of SOS incidence through study completion.	Until the end of the study evaluation, day 180