Sinusoidal Obstruction SyndromeSOSBiomarkerNCT03132337
Sinusoidal Obstruction Syndrome for Stem Cell Transplant Patients Biomarker Study
An observational study of Sinusoidal Obstruction Syndrome, run by Indiana University at 4 sites in United States.
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. Read more about this study
- Who is it for?
- Ages Up to 25 years
- Conditions the study is for
- Sinusoidal Obstruction Syndrome
- What is being tested?
- Serial Blood Draws
- How many people
- 80 participants
- Where
- 4 sitesUnited States
- Study dates
- 1 Apr 2017to 14 Jan 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Stem Cell Transplant patients
Who may be able to join
- Age ≤ 25 years undergoing HCT for any reason who fulfill any ONE (1) of the following criteria:
- History of hepatic disease as defined by:
- Viral hepatitis (i.e., hepatitis C virus [HCV])
- Liver tumor before HCT
- Hepatic fibrosis or cirrhosis before HCT as proven by liver biopsy
- High aspartate aminotransferase (AST) (> 2x ULN) before HCT (pre-transplant evaluation)
Show all 21Show fewer
- High alanine transaminase (ALT) (> 2x ULN) before HCT
- High bilirubin (> 1.2x ULN) before HCT
- HCT high-risk features including:
- 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
- High-risk disease states including:
- Juvenile myelo-monocytic chronic leukemia (JMML)
- Primary hemophagocytic lymphohistiocytosis (HLH)
- Adrenoleukodystrophy
- Osteopetrosis
- Other high-risk features including:
- Prior treatment with gemtuzumab ozogamicin
- Use of hepatotoxic drugs 1 month before HCT and during HCT
- Iron overload (i.e., thalassemia/sickle cell) with serum ferritin > 1000ng/ml
- 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)
- Young age < 2 years but more than 1 month
Who cannot join
- Patients who are transplanted but do not fulfill any of the above mentioned criteria.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
University of Michigan
Ann Arbor, Michigan, 48109
Baylor College of Medicine
Houston, Texas, 77030
Indiana University
Indianapolis, Indiana, 46202
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: Biomarkers of Endothelial Dysfunction in Pediatric Patients Receiving High Intensity Chemotherapy/Irradiation
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.
Read the 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.
- Conditions
- Sinusoidal Obstruction Syndrome
- Being tested
- Serial Blood Draws
- Medical subject headings
- Hepatic Veno-Occlusive Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Serial Blood DrawsOther
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.
| Study group | Role | What they receive |
|---|---|---|
| Stem Cell Transplant | Serial Blood Draws Receives: Other: Serial Blood Draws |
What is being measured
The primary outcome is the main question the study is designed to answer.
SOS proteomic markers
Measure for 3 SOS proteomic markers, L-Ficolin, HA, and ST2, as early predictors of SOS incidence through study completion.
Measured: Until the end of the study evaluation, day 180
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2017
- Study start1 Apr 2017
- Primary completion14 Jan 2021
- Study completion14 Jan 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sinusoidal Obstruction SyndromeStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Sophie Paczesny, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03132337
- Sponsor's protocol ID
- 1701020549
- Acronym
- SOSBiomarker
- Status last verified
- Dec 2023
- First submitted
- 24 Apr 2017
- Last updated
- 19 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 37071469
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Biomarkers of Endothelial Dysfunction in Pediatric Patients Receiving High Intensity Chemotherapy/Irradiation
- ClinicalTrials.gov ID
- NCT03132337
- Acronym / sponsor ID
- SOSBiomarker / 1701020549
- Conditions
- Sinusoidal Obstruction Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Study start
- 1 Apr 2017 (actual)
- Primary completion
- 14 Jan 2021 (actual)
- Study completion
- 14 Jan 2021 (actual)
- First posted
- 27 Apr 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Stem Cell Transplant | – | Other: Serial Blood DrawsSerial Blood Draws |
Who may qualify
- Ages
- Up to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Stem Cell Transplant patients
Key inclusion criteria (21 listed)
- Age ≤ 25 years undergoing HCT for any reason who fulfill any ONE (1) of the following criteria:
- History of hepatic disease as defined by:
- HCT high-risk features including:
- High-risk disease states including:
- Other high-risk features including:
Key exclusion criteria (1 listed)
- Patients who are transplanted but do not fulfill any of the above mentioned criteria.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Serial Blood DrawsDay 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. | Other | Stem Cell Transplant |
Primary outcome
SOS proteomic markers
Time frame: 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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2023.
- Derived from this study: PubMed 37071469Han 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.
- Individual participant dataSharing planned: No
Related trials for Sinusoidal Obstruction Syndrome
Not yet recruitingPhase 2
SAMe in Prevention of Oxaliplatin-associated Liver Injury
Colorectal Cancer, Liver Metastases
RecruitingObservational study
A Prospective Registry of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS).
Hematopoietic Cell Transplantation
RecruitingPhase 2
Defibrotide Dose-escalation for SOS Post-HSCT
Sinusoidal Obstruction Syndrome, Veno-occlusive Disease
RecruitingObservational study
Cell Free DNA Profiling As a Tool to Monitor Clinically-Relevant Events in Allogeneic Hematopoietic Stem Cell Transplantation
GVHD - Graft-Versus-Host Disease, Infections After HSCT
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sinusoidal Obstruction Syndrome, run by Indiana University at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
SOS proteomic markers (time frame: Until the end of the study evaluation, day 180). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03132337, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Dec 2023.