GM-CSF for Reversal of immunopAralysis in pediatriC sEpsis-induced MODS Study
NCT ID: NCT03769844
Last Updated: 2023-02-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE4
120 participants
INTERVENTIONAL
2018-12-05
2023-12-05
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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IV GM-CSF 125 mcg/m2/dose
Subjects in this arm who demonstrate immunoparalysis will receive GM-CSF by the intravenous (IV) route at a dose of 125 mcg/m2/day for 7 consecutive days.
GM-CSF
Subjects demonstrating immunoparalysis (defined by a whole blood ex vivo LP-induced TNF-alpha production capacity \< 200 pg/ml) will receive 7 days of GM-CSF treatment by either the IV or SQ route at a dose of either 125 or 250 mcg/m2/day for 7 days.
SQ GM-CSF 125 mcg/m2/dose
Subjects in this arm who demonstrate immunoparalysis will receive GM-CSF by the subcutaneous (SQ) route at a dose of 125 mcg/m2/day for 7 consecutive days.
GM-CSF
Subjects demonstrating immunoparalysis (defined by a whole blood ex vivo LP-induced TNF-alpha production capacity \< 200 pg/ml) will receive 7 days of GM-CSF treatment by either the IV or SQ route at a dose of either 125 or 250 mcg/m2/day for 7 days.
IV GM-CSF 250 mcg/m2/dose
If the IV 125 mcg/m2/dose arm is not successful in the first cohort of subjects, we will transition to 250 mcg/m2/day via the IV route for 7 consecutive days in a subsequent cohort.
GM-CSF
Subjects demonstrating immunoparalysis (defined by a whole blood ex vivo LP-induced TNF-alpha production capacity \< 200 pg/ml) will receive 7 days of GM-CSF treatment by either the IV or SQ route at a dose of either 125 or 250 mcg/m2/day for 7 days.
SQ GM-CSF 250 mcg/m2/dose
If the SQ 125 mcg/m2/dose arm is not successful in a cohort of subjects (or if the IV dose had to be escalated to 250 mcg/m2/dose), we will transition to 250 mcg/m2/day via the SQ route for 7 consecutive days in a subsequent cohort.
GM-CSF
Subjects demonstrating immunoparalysis (defined by a whole blood ex vivo LP-induced TNF-alpha production capacity \< 200 pg/ml) will receive 7 days of GM-CSF treatment by either the IV or SQ route at a dose of either 125 or 250 mcg/m2/day for 7 days.
Interventions
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GM-CSF
Subjects demonstrating immunoparalysis (defined by a whole blood ex vivo LP-induced TNF-alpha production capacity \< 200 pg/ml) will receive 7 days of GM-CSF treatment by either the IV or SQ route at a dose of either 125 or 250 mcg/m2/day for 7 days.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
17 Years
ALL
No
Sponsors
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Nationwide Children's Hospital
OTHER
Responsible Party
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Mark Hall
Professor, Department of Pediatrics
Principal Investigators
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Mark W Hall, MD
Role: PRINCIPAL_INVESTIGATOR
Nationwide Children's Hospital
Locations
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UCLA Mattel Children's Hospital
Los Angeles, California, United States
Benioff Children's Hospital/UCSF
San Francisco, California, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Countries
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Other Identifiers
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GRACE
Identifier Type: -
Identifier Source: org_study_id
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