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NCT Number: NCT05090345

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

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.

Recruiting

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Key information

Age range

0 year–26 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Mayo Clinic, Rochester, Minnesota, United States

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About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Biorepository Trial

Other

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

Primary outcomes

  1. 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

  2. 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

  3. 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

  4. 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 outcomes

  1. Overall survival.

    Time frame: through study completion, an average of 1 year

Study contacts

Contact information is provided by the study sponsor or research team.

LaTarsha Spencer

CONTACT

[email protected]

(919) 613-1895

Sponsors and collaborators

Lead sponsor

Duke University

Other

Registry information

Official study 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)

Important dates

Study start
2019
Primary completion
2027
Study completion
2028
First posted
Oct 22, 2021
Registry last updated
Jun 1, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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