Clinical trial NCT05594251: Early Detection of Endotheliopathy Post-Transplant
Official title: Early Detection of Endotheliopathy Post-Transplant
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Endotheliopathy
Keywords: Endotheliopathy
Early Detection
Post-Transplant
Lead sponsor: St. Jude Children's Research Hospital
Sponsor class: OTHER
Collaborators: name: Le Bonheur Children's Hospital
name: University of Tennessee
name: Baylor College of Medicine
First posted: 2022-10-26
Enrollment: 32
Sex: ALL
Maximum age (years): 26
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants who meet the Eligibility Criteria

Brief summary: The goal of this research study is to learn more about possible risk factors that might be associated with side effects from stem cell transplants in people between the ages of 0 to 26 years old. Specifically, this study is looking at complications that arise from injury to the endothelium, a small layer of cells lining the blood vessels and heart. These complications can affect the heart, lungs, liver, kidneys and intestines and increase risk of severe illness needing care in the intensive care unit.

Detailed description: Primary Objectives:

* To evaluate the proportion of pediatric patients undergoing Hematopoietic cell transplantation (HCT) who develop critical illness (defined by admission to the pediatric intensive care unit at St. Jude for reasons other than observation after procedure) within 100 days of such therapies.
* To evaluate the proportion of pediatric patients undergoing HCT who experience development of endothelial-related organ dysfunction syndromes.

Secondary Objectives

* To report the baseline (pre-conditioning and post-conditioning but pre-cellular therapy) levels of specified circulating biomarkers, vascular reactivity as measured by reactive hyperemia index (RHI), and to describe baseline clot structure in patients age 0-26 years of age planned to undergo HCT.
* To compare the levels of circulating biomarkers between the cohort of patients that experience critical illness (defined by admission to the pediatric intensive care unit at St. Jude for reasons other than observation after procedure) any time during the first 100 days of HCT following cellular infusion and the cohort of patients that do not ever experience critical illness in the first 100 days of HCT following cellular infusion.
* To evaluate the time to development of any complications including development of endotheliopathies, development of critical illness (defined by admission to the pediatric intensive care unit at St. Jude for reasons other than observation after procedure), development of abnormal vascular reactivity by RHI, development of abnormal clot structure, and time to peak (or trough, if applicable) level of circulating biomarkers.

The study will require a minimum of 1 blood draw before transplant and 6 blood draws throughout the first 100 days following transplant. If participants are admitted to the intensive care unit or are diagnosed with specific complications additional blood draws will be done. Peripheral arterial tonometry (PAT) testing will occur 1-2 times during the first 100 days in several older patients who can tolerate the procedure by sitting still for the duration of the testing period.

Eligibility criteria: Inclusion Criteria:

* Age 0-26 years planned to undergo allogeneic or autologous HCT for any indication with any preparative regimen planned.

Exclusion Criteria:

* Less than 10 kg at time of consent or any child in which blood volume required for scheduled blood draws would pose more than minimal risk.
* Lack of agreement from primary HCT physician.
* Inability or unwillingness of research participant and/or legal guardian/representative to give written informed consent.

Primary outcomes:
measure: Proportion of pediatric patients undergoing Hematopoietic cell transplantation (HCT) who develop critical illness
timeFrame: Up to 100 days after transplant (plus or minus 3 days)
measure: Proportion of pediatric patients undergoing HCT who experience development of endothelial-related organ dysfunction syndromes
timeFrame: Up to 100 days after transplant (plus or minus 3 days)
Secondary outcomes:
measure: Baseline levels of circulating biomarkers
timeFrame: Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)
measure: Baseline vascular reactivity
timeFrame: Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)
measure: Baseline clot structure
timeFrame: Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)
measure: Difference in circulating biomarkers
timeFrame: From Baseline blood draws up to 100 days post ICU admission
measure: Difference in clot structure
timeFrame: From Baseline blood draws up to 100 days post ICU admission
measure: Difference in vascular reactivity
timeFrame: From Baseline blood draws up to 100 days post ICU admission
measure: Time to development of any complications or critical illness
timeFrame: Up to 100 days after transplant (plus or minus 3 days)
Other outcomes:
Locations:
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
Study officials:
affiliation: St. Jude Children's Research Hospital
name: Saad Ghafoor, MD
role: PRINCIPAL_INVESTIGATOR
References: