Phase 4
N=23
GM-CSF for Reversal of immunopAralysis in pediatriC sEpsis-induced MODS Study
Pediatric Sepsis-induced MODS
Bottom Line
View on ClinicalTrials.gov: NCT03769844 ↗Enrolled (actual)
23
Serious AEs
10.5%
Results posted
May 2026
Primary outcome: Primary: Number of Subjects With Restoration of the TNF-alpha Response — 10; 8 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- GM-CSF (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Nationwide Children's Hospital
- Primary completion
- Dec 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects With Restoration of the TNF-alpha Response |
10; 8 | — |
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) 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.
Eligibility Criteria
Inclusion Criteria
- >= 40 weeks gestational age to =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.
Exclusion Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT03769844). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.