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

Hematopoietic Cell Transplantation Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05090345
• Other study ID #: Pro00112697
• Secondary ID: NCI-2021-11324
• Status: Recruiting
• Start date: November 19, 2019
• Est. completion date: February 5, 2026

Study information

• Verified date: March 2024
• Source: Duke University
• Contact: LaTarsha Spencer
• Phone: (919) 613-1895
• Email: latarsha.spencer[@]duke.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: February 5, 2026
• Est. primary completion date: February 5, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 26 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hematopoietic Cell Transplantation
• Hepatic Veno-Occlusive Disease

Intervention

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

Locations

Country	Name	City	State
United States	Duke University	Durham	North Carolina
United States	MD Anderson Cancer Center	Houston	Texas
United States	Mayo Clinic	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Duke University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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	Overall survival.		through study completion, an average of 1 year

External Links

•   Duke University Medical Center