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

NCT05090345 | Recruiting

• 2 other identifiers: Pro00112697NCI-2021-11324
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 3

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.

Conditions

Hematopoietic Cell Transplantation

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (1)

• Overall survival.

  through study completion, an average of 1 year

Study Arms (1)

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.

Other: Biorepository Trial

Interventions

Biorepository Trial OTHER

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

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

Eligibility Criteria

• Age: 0 Years - 26 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

Children, adolescents and young adults (AYA) 0-26 years of age undergoing HSCT.

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Duke University: lead

Study Sites (3)

Mayo Clinic

Rochester, Minnesota, 55902, United States

RECRUITING

Duke University

Durham, North Carolina, 27705, United States

RECRUITING

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• Duke University Medical Center

Study Officials

• Kris Mahadeo

  Duke University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

LaTarsha Spencer

CONTACT

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 12, 2021
• First Posted: October 22, 2021
• Study Start: November 19, 2019
• Primary Completion: February 5, 2026
• Study Completion: February 5, 2026
• Last Updated: April 20, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)