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

Recruiting

• Conditions: Hematopoietic Cell Transplantation
• Interventions: Other: Biorepository Trial

• Registration Number: NCT05090345
• Lead Sponsor: Duke University

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).

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2019-11-19
• Study First Submitted: 2021-10-12
• Study First Submitted QC: 2021-10-12
• Study First Posted: 2021-10-22
• Status Verified: 2024-03
• Last Update Submitted: 2024-03-12
• Last Update Posted: 2024-03-13
• Primary Completion: 2026-02-05
• Study Completion: 2026-02-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 500

Inclusion Criteria

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

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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.

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Biorepository trial of children and adolescents/young adults (AYA)	Biorepository Trial	undergoing hematopoietic cell transplantation (HCT) to assess the impact of endotheliopathies in the HCT setting as a contributor of significant morbidity and mortality.

Primary Outcome Measures

Name	Time	Method
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 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 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 inflammatory factors within 100 days post HCT-CT in the setting of endotheliopathies.	through study completion, an average of 1 year

Secondary Outcome Measures

Name	Time	Method
Overall survival.	through study completion, an average of 1 year

Trial Locations

Locations (3)

MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States

Duke University; 🇺🇸 Durham, North Carolina, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States