GM-CSF for Reversal of immunopAralysis in pediatriC sEpsis-induced MODS Study (NCT03769844) | Clinical Trial Compass
CompletedPhase 4
GM-CSF for Reversal of immunopAralysis in pediatriC sEpsis-induced MODS Study
United States75 participantsStarted 2018-12-05
Plain-language 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.
Who can participate
Age range
17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* \>= 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.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Subjects With Restoration of the TNF-alpha Response
Timeframe: Subjects will be screened for immunoparalysis throughout their first three weeks of sepsis-induced MODS