EndotheliopathyNCT05594251
Early Detection of Endotheliopathy Post-Transplant
An observational study of Endotheliopathy, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 26 years
- Conditions the study is for
- Endotheliopathy
- How many people
- 32 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Nov 2022to 5 May 2024
- 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 26 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Participants who meet the Eligibility Criteria
Who may be able to join
- Age 0-26 years planned to undergo allogeneic or autologous HCT for any indication with any preparative regimen planned.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
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.
Read the 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.
- Conditions
- Endotheliopathy
- Keywords
- Endotheliopathy, Early Detection, Post-Transplant
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Up to 100 days after transplant (plus or minus 3 days)
Secondary outcomes (7)
Baseline levels of circulating biomarkers
Descriptive statistics (mean, median, standard deviation) will be used.
Measured: Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)
Baseline vascular reactivity
Descriptive statistics (mean, median, standard deviation) will be used.
Measured: Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)
Baseline clot structure
Descriptive statistics (mean, median, standard deviation) will be used.
Measured: Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)
Difference in circulating biomarkers
We will compare the levels of circulating biomarkers 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) and the patients that do not ever experience critical illness by using the linear mixed-effect model (LMM). Blood draws will be done at baseline, Day 0, and after transplant: days 1, 7, 14, 21, 28, 100 (plus or minus 3 days). Those with Endotheliopathy diagnosis anytime during the 100 days of HCT following cellular transfusion, and are admitted to ICU, will have additional blood draws: At the time of Endotheliopathy diagnosis (plus or minus 3 days), at the time of ICU admission (plus or minus 3 days), 72 hours after ICU admission (plus or minus 3 days), and every 7 days if remains admitted to the intensive care unit for longer than 72 hours (plus or minus 3 days) up to 100 days post ICU submission.
Measured: From Baseline blood draws up to 100 days post ICU admission
Difference in clot structure
We will compare the clot structure 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) and the patients that do not ever experience critical illness by using the linear mixed-effect model (LMM). Blood draws will be done at baseline, Day 0, and after transplant: days 1, 7, 14, 21, 28, 100 (plus or minus 3 days). Those with Endotheliopathy diagnosis anytime during the 100 days of HCT following cellular transfusion, and are admitted to ICU, will have additional blood draws: At the time of Endotheliopathy diagnosis (plus or minus 3 days), at the time of ICU admission (plus or minus 3 days), 72 hours after ICU admission (plus or minus 3 days), and every 7 days if remains admitted to the intensive care unit for longer than 72 hours (plus or minus 3 days) up to 100 days post ICU submission.
Measured: From Baseline blood draws up to 100 days post ICU admission
Difference in vascular reactivity
We will compare the vascular reactivity 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) and the patients that do not ever experience critical illness by using the linear mixed-effect model (LMM). Blood draws will be done at baseline, Day 0, and after transplant: days 1, 7, 14, 21, 28, 100 (plus or minus 3 days). Those with Endotheliopathy diagnosis anytime during the 100 days of HCT following cellular transfusion, and are admitted to ICU, will have additional blood draws: At the time of Endotheliopathy diagnosis (plus or minus 3 days), at the time of ICU admission (plus or minus 3 days), 72 hours after ICU admission (plus or minus 3 days), and every 7 days if remains admitted to the intensive care unit for longer than 72 hours (plus or minus 3 days) up to 100 days post ICU submission.
Measured: From Baseline blood draws up to 100 days post ICU admission
Time to development of any complications or critical illness
Will be assessed by the Kaplan-Meier method
Measured: Up to 100 days after transplant (plus or minus 3 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2022
- Study start28 Nov 2022
- Primary completion5 May 2024
- Study completion5 May 2024
Why it stopped: IRB approval 1/6/2025 to close to accrual with present enrollment. Project completed.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for EndotheliopathyStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe 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
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Le Bonheur Children's Hospital, University of Tennessee, Baylor College of Medicine
- Responsible party
- Sponsor
- Investigators
- Saad Ghafoor, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05594251
- Sponsor's protocol ID
- EDEPT
- Status last verified
- Jan 2025
- First submitted
- 14 Oct 2022
- Last updated
- 24 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Early Detection of Endotheliopathy Post-Transplant
- ClinicalTrials.gov ID
- NCT05594251
- Acronym / sponsor ID
- EDEPT
- Conditions
- Endotheliopathy
- Keywords
- Endotheliopathy, Early Detection, Post-Transplant
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: IRB approval 1/6/2025 to close to accrual with present enrollment. Project completed.
- Enrolment
- 32 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Collaborators
- Le Bonheur Children's Hospital, University of Tennessee, Baylor College of Medicine
- Study start
- 28 Nov 2022 (actual)
- Primary completion
- 5 May 2024 (actual)
- Study completion
- 5 May 2024 (actual)
- First posted
- 26 Oct 2022 (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 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Participants who meet the Eligibility Criteria
Key inclusion criteria (1 listed)
- Age 0-26 years planned to undergo allogeneic or autologous HCT for any indication with any preparative regimen planned.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Proportion of pediatric patients undergoing Hematopoietic cell transplantation (HCT) who develop critical illness
Time frame: Up to 100 days after transplant (plus or minus 3 days)
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.
Proportion of pediatric patients undergoing HCT who experience development of endothelial-related organ dysfunction syndromes
Time frame: Up to 100 days after transplant (plus or minus 3 days)
The proportion will be estimated using one-sample binomial estimates and various confidence intervals.
7 secondary outcomes
Baseline levels of circulating biomarkers
Time frame: Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)
Baseline vascular reactivity
Time frame: Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)
Baseline clot structure
Time frame: Baseline (before preparative transplant regimen starts) and Day 0 (on day of transplant, before therapy takes place)
Difference in circulating biomarkers
Time frame: From Baseline blood draws up to 100 days post ICU admission
Difference in clot structure
Time frame: From Baseline blood draws up to 100 days post ICU admission
Difference in vascular reactivity
Time frame: From Baseline blood draws up to 100 days post ICU admission
Time to development of any complications or critical illness
Time frame: Up to 100 days after transplant (plus or minus 3 days)
Results and safety
The study was stopped early (reason given: IRB approval 1/6/2025 to close to accrual with present enrollment. Project completed.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jan 2025.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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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.
Saad Ghafoor, MD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Endotheliopathy, run by St. Jude Children's Research Hospital at 1 site 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 1 inclusion and 3 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: stopped early, as of the sponsor's last update on 24 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 was stopped early (reason given: IRB approval 1/6/2025 to close to accrual with present enrollment. Project completed.). Results are not available in the source record.
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?
Proportion of pediatric patients undergoing Hematopoietic cell transplantation (HCT) who develop critical illness (time frame: Up to 100 days after transplant (plus or minus 3 days)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 24 Jan 2025.