A Prospective Registry of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS).

Prospective Multi Center Study of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS)

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05090345

Enrollment

500

Registered

2021-10-22

Start date

2019-11-19

Completion date

2028-02-05

Last updated

2026-06-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hematopoietic Cell Transplantation

Brief 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.

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).

Interventions

OTHERBiorepository Trial

participating in this arm, peripheral blood specimen(s) will be obtained at 8 specified time-points.

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

0 Years to 26 Years

Healthy volunteers

Inclusion criteria

* Children, adolescents and young adults (AYA) 0-26 years of age undergoing HSCT. * Recipients of autologous and allogeneic HSCT. * Any preparative regimen.

Exclusion criteria

* 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.

Design outcomes

Primary

Measure	Time frame
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.	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.	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.	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.	through study completion, an average of 1 year

Secondary

Measure	Time frame
Overall survival.	through study completion, an average of 1 year

Countries

United States

Contacts

CONTACTLaTarsha Spencer

latarsha.spencer@duke.edu(919) 613-1895

PRINCIPAL_INVESTIGATORKris Mahadeo

Duke University

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jun 2, 2026