Hematopoietic Cell TransplantationNCT05090345
A Prospective Registry of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS).
An observational study of Hematopoietic Cell Transplantation, run by Duke University at 3 sites in United States.
Trial summary
This is a prospective international multi-center registry and biorepository trial of children and adolescents/young adults (AYA) undergoing hematopoietic cell transplantation (HCT) to assess the impact of endotheliopathies in the HCT setting as a contributor of significant morbidity and mortality. Read more about this study
- Who is it for?
- Ages 0 to 26 years
- Conditions the study is for
- Hematopoietic Cell Transplantation
- What is being tested?
- Biorepository Trial
- How many people
- 500 participants
- Where
- 3 sitesUnited States
- Study dates
- 19 Nov 2019to 5 Feb 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 0 to 26 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children, adolescents and young adults (AYA) 0-26 years of age undergoing HSCT.
Who may be able to join
- Children, adolescents and young adults (AYA) 0-26 years of age undergoing HSCT.
- Recipients of autologous and allogeneic HSCT.
- Any preparative regimen.
Who cannot join
- Any patient who does not consent/assent to participation.
- Any patient for whom 5 mL blood sample(s) drawn at specified intervals would pose any more than minimal risk, as defined by institutional guidelines and at discretion of treating physician will be ineligible for biorepository banking.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites, 3 recruiting
- Recruiting
Duke University
Durham, North Carolina, 27705
Kris Mahadeo, 919-668-1180, kris.mahadeo@duke.edu
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Dristhi Ragoonanan, DRagoonanan@mdanderson.org
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55902
Mira Kohorst, kohorst.mira@mayo.edu
About this study
Official title: Prospective Multi Center Study of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS)
This is a prospective international multi-center registry and biorepository trial of children and adolescents/young adults (AYA) undergoing hematopoietic cell transplantation (HCT) to assess the impact of endotheliopathies in the HCT setting as a contributor of significant morbidity and mortality.
Read the detailed description
Primary:
- To evaluate the proportion of pediatric patients undergoing hematopoietic cell transplant (HCT) who develop endotheliopathies (SOS, TMA, DAH and IPS) and/or MODS within 100 days of such therapies.
- To evaluate the proportion of pediatric patients undergoing hematopoietic cell transplant (HCT) who experience abnormal levels of circulating angiogenic, vascular permeability and inflammatory factors within 100 days post HCT-CT in the setting of endotheliopathies.
Secondary/Exploratory Objective(s):
- To determine the impact of endotheliopathies in the HCT setting on the overall survival.
- To assess the efficacy of specific prophylaxis and treatment strategies directed at endotheliopathies (SOS, TMA, DAH and IPS) post-HCT among enrolled children on overall survival.
- To assess whether exposure to immunotherapies (CAR-T, PD-1 Inhibitors, bispecific T-cell engager (BiTE) antibodies and antibody drug conjugates) prior to HCT affects post-HCT outcomes (endotheliopathies and MODS).
- Conditions
- Hematopoietic Cell Transplantation
- Being tested
- Biorepository Trial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biorepository TrialOther
participating in this arm, peripheral blood specimen(s) will be obtained at 8 specified time-points.
| Study group | Role | What they receive |
|---|---|---|
| Biorepository trial of children and adolescents/young adults (AYA) | undergoing hematopoietic cell transplantation (HCT) to assess the impact of endotheliopathies in the HCT setting as a contributor of significant morbidity and mortality. Receives: Other: Biorepository Trial |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of pediatric patients undergoing hematopoietic cell transplant (HCT) who develop endotheliopathies (SOS, TMA, DAH and IPS) and/or MODS within 100 days of such therapies.
Measured: through study completion, an average of 1 year
Proportion of pediatric patients undergoing (HCT) who experience abnormal levels of circulating angiogenic factors within 100 days post HCT-CT in the setting of endotheliopathies.
Measured: through study completion, an average of 1 year
Proportion of pediatric patients undergoing (HCT) who experience abnormal levels of vascular permeability within 100 days post HCT-CT in the setting of endotheliopathies.
Measured: through study completion, an average of 1 year
Proportion of pediatric patients undergoing (HCT) who experience abnormal levels of inflammatory factors within 100 days post HCT-CT in the setting of endotheliopathies.
Measured: through study completion, an average of 1 year
Secondary outcomes (1)
Overall survival.
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2021
- Study start19 Nov 2019
- Primary completion5 Feb 2027 (estimated)
- Study completion5 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic Cell TransplantationStudies of the same condition, open and under way.
- More trials run by Duke 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
- 500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kris Mahadeo, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05090345
- Sponsor's protocol ID
- Pro00112697
- Other IDs
- NCI-2021-11324
- Status last verified
- May 2026
- First submitted
- 12 Oct 2021
- Last updated
- 1 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Multi Center Study of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS)
- ClinicalTrials.gov ID
- NCT05090345
- Acronym / sponsor ID
- Pro00112697
- Conditions
- Hematopoietic Cell Transplantation
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Study start
- 19 Nov 2019 (actual)
- Primary completion
- 5 Feb 2027 (estimated)
- Study completion
- 5 Feb 2028 (estimated)
- First posted
- 22 Oct 2021 (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.
This is a prospective international multi-center registry and biorepository trial of children and adolescents/young adults (AYA) undergoing hematopoietic cell transplantation (HCT) to assess the impact of endotheliopathies in the HCT setting as a contributor of significant morbidity and mortality.
Detailed description
Primary:
- To evaluate the proportion of pediatric patients undergoing hematopoietic cell transplant (HCT) who develop endotheliopathies (SOS, TMA, DAH and IPS) and/or MODS within 100 days of such therapies.
- To evaluate the proportion of pediatric patients undergoing hematopoietic cell transplant (HCT) who experience abnormal levels of circulating angiogenic, vascular permeability and inflammatory factors within 100 days post HCT-CT in the setting of endotheliopathies.
Secondary/Exploratory Objective(s):
- To determine the impact of endotheliopathies in the HCT setting on the overall survival.
- To assess the efficacy of specific prophylaxis and treatment strategies directed at endotheliopathies (SOS, TMA, DAH and IPS) post-HCT among enrolled children on overall survival.
- To assess whether exposure to immunotherapies (CAR-T, PD-1 Inhibitors, bispecific T-cell engager (BiTE) antibodies and antibody drug conjugates) prior to HCT affects post-HCT outcomes (endotheliopathies and MODS).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Biorepository trial of children and adolescents/young adults (AYA) | – | Other: Biorepository Trialundergoing hematopoietic cell transplantation (HCT) to assess the impact of endotheliopathies in the HCT setting as a contributor of significant morbidity and mortality. |
Who may qualify
- Ages
- 0 to 26 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children, adolescents and young adults (AYA) 0-26 years of age undergoing HSCT.
Key inclusion criteria (3 listed)
- Children, adolescents and young adults (AYA) 0-26 years of age undergoing HSCT.
- Recipients of autologous and allogeneic HSCT.
- Any preparative regimen.
Key exclusion criteria (2 listed)
- Any patient who does not consent/assent to participation.
- Any patient for whom 5 mL blood sample(s) drawn at specified intervals would pose any more than minimal risk, as defined by institutional guidelines and at discretion of treating physician will be ineligible for biorepository banking.
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 |
|---|---|---|
| Biorepository Trialparticipating in this arm, peripheral blood specimen(s) will be obtained at 8 specified time-points. | Other | Biorepository trial of children and adolescents/young adults (AYA) |
Primary outcome
Proportion of pediatric patients undergoing hematopoietic cell transplant (HCT) who develop endotheliopathies (SOS, TMA, DAH and IPS) and/or MODS within 100 days of such therapies.
Time frame: through study completion, an average of 1 year
Proportion of pediatric patients undergoing (HCT) who experience abnormal levels of circulating angiogenic factors within 100 days post HCT-CT in the setting of endotheliopathies.
Time frame: through study completion, an average of 1 year
Proportion of pediatric patients undergoing (HCT) who experience abnormal levels of vascular permeability within 100 days post HCT-CT in the setting of endotheliopathies.
Time frame: through study completion, an average of 1 year
Proportion of pediatric patients undergoing (HCT) who experience abnormal levels of inflammatory factors within 100 days post HCT-CT in the setting of endotheliopathies.
Time frame: through study completion, an average of 1 year
1 secondary outcome
Overall survival.
Time frame: through study completion, an average of 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2026.
- Duke University Medical CenterLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Kris Mahadeo principal investigator
Duke University
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hematopoietic Cell Transplantation, run by Duke University at 3 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 3 inclusion and 2 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: looking for participants now, as of the sponsor's last update on 1 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact LaTarsha Spencer ((919) 613-1895, latarsha.spencer@duke.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of pediatric patients undergoing hematopoietic cell transplant (HCT) who develop endotheliopathies (SOS, TMA, DAH and IPS) and/or MODS within 100 days of such therapies. (time frame: through study completion, an average of 1 year). The study lists 4 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 1 Jun 2026.