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

Last updated: April 16, 2025
Sponsor: Duke University
Overall Status: Active - Recruiting

Phase

N/A

Condition

Bone Marrow Transplant

Treatment

Biorepository Trial

Clinical Study ID

NCT05090345
Pro00112697
NCI-2021-11324
  • Ages < 26
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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

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 poseany more than minimal risk, as defined by institutional guidelines and at discretionof treating physician will be ineligible for biorepository banking.

Study Design

Total Participants: 500
Treatment Group(s): 1
Primary Treatment: Biorepository Trial
Phase:
Study Start date:
November 19, 2019
Estimated Completion Date:
February 05, 2026

Study 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).

Connect with a study center

  • Mayo Clinic

    Rochester, Minnesota 55902
    United States

    Active - Recruiting

  • Duke University

    Durham, North Carolina 27705
    United States

    Active - Recruiting

  • M D Anderson Cancer Center

    Houston, Texas 77030
    United States

    Active - Recruiting

  • MD Anderson Cancer Center

    Houston, Texas 77030
    United States

    Active - Recruiting

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