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

NCT ID: NCT05090345

Last Updated: 2025-04-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

500 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-11-19

Study Completion Date

2026-02-05

Brief Summary

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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

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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

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Hematopoietic Cell Transplantation

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

Biorepository Trial

Intervention Type OTHER

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

Interventions

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Biorepository Trial

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

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

0 Years

Maximum Eligible Age

26 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Duke University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Kris Mahadeo

Role: PRINCIPAL_INVESTIGATOR

Duke University

Locations

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Mayo Clinic

Rochester, Minnesota, United States

Site Status RECRUITING

Duke University

Durham, North Carolina, United States

Site Status RECRUITING

MD Anderson Cancer Center

Houston, Texas, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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LaTarsha Spencer

Role: CONTACT

(919) 613-1895

Facility Contacts

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Mira Kohorst

Role: primary

Kris Mahadeo

Role: primary

919-668-1180

Dristhi Ragoonanan

Role: primary

Related Links

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http://www.dukehealth.org

Duke University Medical Center

Other Identifiers

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NCI-2021-11324

Identifier Type: OTHER

Identifier Source: secondary_id

Pro00112697

Identifier Type: -

Identifier Source: org_study_id

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