GM-CSF for Reversal of immunopAralysis in pediatriC sEpsis-induced MODS Study
Status:
Recruiting
Trial end date:
2021-12-05
Target enrollment:
Participant gender:
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.