MedDataX Logo

Sinusoidal Obstruction Syndrome for Stem Cell Transplant Patients Biomarker Study

NCT ID: NCT03132337

Last Updated: 2023-12-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

80 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-04-01

Study Completion Date

2021-01-14

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Sinusoidal Obstruction Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Stem Cell Transplant

Serial Blood Draws

Serial Blood Draws

Intervention Type OTHER

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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Maximum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Indiana University

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Sophie Paczesny

Professor, Department of Pediatrics

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

Indiana University

Indianapolis, Indiana, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

Han Y, Bidgoli A, DePriest BP, Mendez A, Bijangi-Vishehsaraei K, Perez-Albuerne ED, Krance RA, Renbarger J, Skiles JL, Choi SW, Liu H, Paczesny S. Prospective assessment of risk biomarkers of sinusoidal obstruction syndrome after hematopoietic cell transplantation. JCI Insight. 2023 May 22;8(10):e168221. doi: 10.1172/jci.insight.168221.

Reference Type DERIVED
PMID: 37071469 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

1701020549

Identifier Type: -

Identifier Source: org_study_id