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

Duke University

Status: Enrolling

Conditions: Hematopoietic Cell Transplantation

Treatments: Other: Biorepository Trial

Study type: Observational

Funder types: Other

Identifiers

NCT05090345

Pro00112697

NCI-2021-11324 (Other Identifier)

Details and patient eligibility

About

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.

Full 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).

Enrollment: 500 estimated patients

Sex: All

Ages: Under 26 years old

Volunteers: No 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.

Trial design

500 participants in 1 patient group

Biorepository trial of children and adolescents/young adults (AYA)

Description:

undergoing hematopoietic cell transplantation (HCT) to assess the impact of endotheliopathies in the HCT setting as a contributor of significant morbidity and mortality.

Treatment:

Other: Biorepository Trial