GM-CSF for Reversal of Immunoparalysis in Pediatric Sepsis-induced MODS

a study on Multiple Organ Dysfunction Syndrome Sepsis

Summary

Eligibility
for people ages up to 17 years (full criteria)
Location
at Oakland, California and other locations
Dates
study started
completion around

Description

Summary

The GRACE-2 study is a prospective, multi-center, double-blind, randomized controlled trial of the drug GM-CSF vs placebo in children with sepsis-induced multiple organ dysfunction syndrome (MODS) who have immunoparalysis with mild to moderate inflammation.

Details

The GRACE-2 study is a is a prospective, multi-center, double-blind, randomized controlled trial of the drug GM-CSF vs placebo in children with sepsis-induced multiple organ dysfunction syndrome (MODS) who have immunoparalysis with mild to moderate inflammation. Eligible subjects will undergo centralized immunophenotyping on MODS Day 2. Those who are found to have immunoparalysis (a whole blood LPS-induced TNF-alpha production capacity < 200 pg/ml) with mild to moderate inflammation (serum ferritin level < 2000 ng/ml) will be randomized to receive intravenous (IV) GM-CSF at a dose of 125 mcg/m2/day x 7 days or placebo. The primary outcome variable is the cumulative 28-day Pediatric Logistic Organ Dysfunction (PELOD)-2 score. Secondary outcomes include measures of health-related quality of life and function status at 3 months from randomization.

Keywords

Pediatric Sepsis-induced Multiple Organ Dysfunction Syndrome, Sepsis, Toxemia, Multiple Organ Failure, GM-CSF

Eligibility

You can join if…

Open to people ages up to 17 years

  • ≥ 40 weeks corrected gestational age to < 18 years; AND
  • Admission to the PICU or CICU; AND
  • Onset of ≥ 2 new organ dysfunctions within the last 3 calendar days (compared to pre-sepsis baseline) as measured by the modified Proulx criteria; AND
  • Documented or suspected infection as the MODS inciting event.

You CAN'T join if...

  • Weight <3kg; OR
  • Limitation of care order at the time of screening; OR
  • Patients at high likelihood of progression to brain death in opinion of the clinical team; OR
  • Moribund condition in which the patient is unlikely to survive the next 48 hours in opinion of the clinical team; OR
  • History of myeloid leukemia, myelodysplasia, or autoimmune thrombocytopenia; OR
  • Current or prior diagnosis of hemophagocytic lymphohistiocytosis or macrophage activation syndrome; OR
  • Peripheral white blood cell count < 1,000 cells/mm3 as the result of myeloablative therapy OR receipt of myeloablative therapy within the previous 14 days; OR
  • Known allergy to GM-CSF; OR
  • Known pregnancy; OR
  • Lactating females; OR
  • Receipt of anakinra or GM-CSF within the previous 28 days; OR
  • Resolution of MODS by MODS Day 2; OR
  • Previous enrollment in the GRACE-2 study.

Locations

  • Benioff Children's Hospital - Oakland accepting new patients
    Oakland California 64609 United States
  • Benioff Children's Hospital - Mission Bay accepting new patients
    San Francisco California 94143 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Nationwide Children's Hospital
ID
NCT05266001
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 400 study participants
Last Updated
Contact us about this trial