Clinical trial NCT05090345: A Prospective Registry of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS).
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)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hematopoietic Cell Transplantation
Lead sponsor: Duke University
Sponsor class: OTHER
First posted: 2021-10-22
Enrollment: 500
Sex: ALL
Minimum age (years): 0
Maximum age (years): 26
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Children, adolescents and young adults (AYA) 0-26 years of age undergoing HSCT.

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).

Eligibility criteria: 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.

Primary outcomes:
measure: 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.
timeFrame: through study completion, an average of 1 year
measure: 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.
timeFrame: through study completion, an average of 1 year
measure: Proportion of pediatric patients undergoing (HCT) who experience abnormal levels of vascular permeability within 100 days post HCT-CT in the setting of endotheliopathies.
timeFrame: through study completion, an average of 1 year
measure: Proportion of pediatric patients undergoing (HCT) who experience abnormal levels of inflammatory factors within 100 days post HCT-CT in the setting of endotheliopathies.
timeFrame: through study completion, an average of 1 year
Secondary outcomes:
measure: Overall survival.
timeFrame: through study completion, an average of 1 year
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
Study officials:
affiliation: Duke University
name: Kris Mahadeo
role: PRINCIPAL_INVESTIGATOR
References: