A Prospective Registry of Pediatric Cellular Therapy Patients at Risk for Endothelial Dysfunction, Sinusoidal Obstruction Syndrome and/or Multi-Organ Dysfunction Syndrome (MODS).
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Biorepository Trial.
- Who it may be relevant to
- Registry conditions: Hematopoietic Cell Transplantation. Basic parameters: 0 years — 26 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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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)
Overview
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).
Interventions
- Other Biorepository Trial
participating in this arm, peripheral blood specimen(s) will be obtained at 8 specified time-points.
Primary outcome measures
- 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. [Time frame: 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. [Time frame: 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. [Time frame: 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. [Time frame: through study completion, an average of 1 year]
Secondary outcome measures (1)
- Overall survival. [Time frame: through study completion, an average of 1 year]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 3 centers
- Mayo Clinic — Rochester
- Duke University — Durham
- MD Anderson Cancer Center — Houston
Identifiers
NCT: NCT05090345 · Pro00112697 · NCI-2021-11324