Early Detection of Endotheliopathy Post-Transplant

NCT05594251 | Terminated

• 1 other identifier: EDEPT
• Study Type: observational
• Target: 32
• Locations: 1 country
• Sites: 1

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.

Conditions

Endotheliopathy

Keywords

Endotheliopathy; Early Detection; Post-Transplant

Outcome Measures

Primary Outcomes (2)

• Proportion of pediatric patients undergoing Hematopoietic cell transplantation (HCT) who develop critical illness

  The proportion of pediatric patients undergoing HCT who develop critical illness (defined by admission to the pediatric intensive care unit at St. Jude for reasons other than observation after procedure) will be estimated using one-sample binomial estimates and various confidence intervals.

  Up to 100 days after transplant (plus or minus 3 days)

• Proportion of pediatric patients undergoing HCT who experience development of endothelial-related organ dysfunction syndromes

  The proportion will be estimated using one-sample binomial estimates and various confidence intervals.

  Up to 100 days after transplant (plus or minus 3 days)

Secondary Outcomes (7)

• Baseline levels of circulating biomarkers

  Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)

• Baseline vascular reactivity

  Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)

• Baseline clot structure

  Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)

• Difference in circulating biomarkers

  From Baseline blood draws up to 100 days post ICU admission

• Difference in clot structure

  From Baseline blood draws up to 100 days post ICU admission

Eligibility Criteria

• Age: Up to 26 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Participants who meet the Eligibility Criteria

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead
• Le Bonheur Children's Hospital: collaborator
• University of Tennessee: collaborator
• Baylor College of Medicine: collaborator

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

• St. Jude Children's Research Hospital
• Clinical Trials Open at St. Jude

Study Officials

• Saad Ghafoor, MD

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 14, 2022
• First Posted: October 26, 2022
• Study Start: November 28, 2022
• Primary Completion: May 5, 2024
• Study Completion: May 5, 2024
• Last Updated: January 24, 2025

Record last verified: 2025-01

Locations

US (1)