NCT05090345

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
• Phase: —
• Study type: Observational
• Enrollment: 500 (estimated)
• Sponsor: Duke University · Academic / Other
• Sex: All
• Age: 0 Years – 26 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Hematopoietic Cell Transplantation

Interventions

Type	Name	Description
OTHER	Biorepository Trial	participating in this arm, peripheral blood specimen(s) will be obtained at 8 specified time-points.

Timeline

• Start date: 2019-11-19
• Primary completion: 2026-02-05
• Completion: 2026-02-05
• First posted: 2021-10-22
• Last updated: 2025-04-20

Locations

3 sites across 1 country: United States