Summary

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

Description

Summary

This study is an open-label, multi-center, interventional trial in which children with sepsis-induced MODS undergo surveillance immune function testing beginning on Day 2 of MODS. Those children who demonstrate immunoparalysis (TNF-alpha response <200 pg/ml) will receive a 7-day course of GM-CSF at a dose of 125 or 250 mcg/m2/day by either the intravenous (IV) or subcutaneous (SQ) route.

The goal of the study is to establish the dose and route of delivery that results in resolution of immunoparalysis (TNF-alpha response >=200 pg/ml) by the morning after the 3rd scheduled dose with persistent resolution of immunoparalysis on the morning after the 7th scheduled dose. Resolution of immunoparalysis in 8 out of the first 10 subjects in a study treatment arm represents a successful dose and route. The goal of this study will be achieved through the following Specific Aims:

Specific Aim 1. Establish the immunologic efficacy of GM-CSF administered by the IV and SQ routes in children with immunoparalysis in the setting of sepsis-induced MODS.

Specific Aim 2. Estimate the pharmacokinetic parameters by the IV and SQ GM-CSF administered in pediatric sepsis-induced MODS.

Specific Aim 3. Demonstrate the feasibility of screening, enrollment, drug delivery, and sample collection for a multi-center immunostimulation trial in children with sepsis-induced MODS.

Official Title

GM-CSF for Reversal of immunopAralysis in pediatriC sEpsis-induced MODS (GRACE)

Keywords

Pediatric Sepsis-induced MODS, sepsis, MODS, pediatric, immunoparalysis, GM-CSF, Toxemia, Molgramostim, Sargramostim

Eligibility

You can join if…

Open to people ages up to 17 years

  • >= 40 weeks gestational age to <18 years; AND
  • Onset of >=2 new organ dysfunctions (compared to pre-sepsis baseline) as measured by the Proulx criteria; AND
  • Documented or suspected infection as the MODS inciting event.

You CAN'T join if...

  • Unable to collect a cumulative total of 20.5 mL of blood for this study due to research blood draw limits; OR
  • Limitation of care order at the time of screening; OR
  • Patients at high risk for brain death; OR
  • Active (or planned within 7 days) immunosuppressive treatment for oncologic, transplant, or rheumatologic disease; OR
  • Known primary immunodeficiency disorder; OR
  • Diagnosis of myeloid leukemia, myelodysplasia, or autoimmune thrombocytopenia;OR
  • Known allergy to GM-CSF; OR
  • Documented hyperferritinemia (serum ferritin >= 500 ng/ml) during current sepsis event; OR
  • Contraindication to SQ injection (ECMO); OR
  • Burns where >5% of the total body surface area is affected; OR
  • Renal replacement therapy at the time of screening; OR
  • On ECMO or anticipated to require ECMO; OR
  • Known pregnancy; OR
  • Inability to collect and ship sample for immune testing on MODS Day 2; OR
  • Previous enrollment in the GRACE study

Locations

  • UCLA Mattel Children's Hospital
    Los Angeles California 90095 United States
  • Benioff Children's Hospital/UCSF
    San Francisco California 94158 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Nationwide Children's Hospital
ID
NCT03769844
Phase
Phase 4 research study
Study Type
Interventional
Participants
Expecting 120 study participants
Last Updated