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GM-CSF for Immunomodulation Following Trauma (GIFT) Study (GIFT)

This study is currently recruiting participants.
Verified July 2017 by Mark Hall, Nationwide Children's Hospital
Sponsor:
ClinicalTrials.gov Identifier:
NCT01495637
First Posted: December 20, 2011
Last Update Posted: July 26, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborators:
National Institute of General Medical Sciences (NIGMS)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Information provided by (Responsible Party):
Mark Hall, Nationwide Children's Hospital
December 13, 2011
December 20, 2011
July 26, 2017
December 2011
June 2018   (Final data collection date for primary outcome measure)
Immune function [ Time Frame: 7-days post-trauma ]
To identify the lowest immunostimulatory yet tolerable dose of GM-CSF that produces lasting improvement in innate immune function in treated children.
Nosocomial infection [ Time Frame: 14-days post-trauma ]
Complete list of historical versions of study NCT01495637 on ClinicalTrials.gov Archive Site
Nosocomial infection [ Time Frame: 28-days post-trauma ]
The development of hospital-acquired infection through post-trauma day 28
  • Innate immune function [ Time Frame: Day 4 post-trauma ]
    To identify the lowest immunostimulatory yet tolerable dose of GM-CSF that produces lasting improvement in innate immune function in treated children.
  • Nosocomial infection [ Time Frame: 28-days post-trauma ]
  • Innate Immune Function [ Time Frame: Day 14 post-trauma ]
    To identify the lowest immunostimulatory yet tolerable dose of GM-CSF that produces lasting improvement in innate immune function in treated children.
Not Provided
Not Provided
 
GM-CSF for Immunomodulation Following Trauma (GIFT) Study
GM-CSF for Immunomodulation Following Trauma (GIFT) Study
The GIFT study is a prospective, multi-center, interventional trial using the drug GM-CSF for the reversal of innate immune suppression in critically injured children. The study will be conducted in two phases, a dose-finding phase then an efficacy phase. The dose-finding phase is the current active phase of the study. The central hypothesis of the study is that immunomodulation with GM-CSF will result in reduction in the risk of nosocomial infection after critical injury in high-risk children through safe, rapid, and sustained improvement in innate immune function.
The current phase of the study is an open-label dose-finding phase in which critically injured children undergo prospective, serial immune function testing in the first few days after injury. If a subject's immune function (as measured by whole blood ex vivo LPS-induced TNF-alpha production capacity) is below a critical threshold, the subject will receive GM-CSF at a dose of 30, 62, or 125 mcg/m2 per day for three days. Enrollment is stratified by pubertal status (Tanner 1 or Tanner > 1) and by presence or absence of severe traumatic brain injury (TBI). Dose-finding is being conducted independently in each of these strata. The outcome variable for the dose-finding phase of the GIFT study is restoration of TNF-alpha production capacity and monocyte HLA-DR expression by the end of treatment, persisting to post-trauma day 7. A subsequent randomized, placebo-controlled trial with nosocomial infection as the primary outcome variable is planned once dose-finding is complete. This study is being conducted by the NICHD's Collaborative Pediatric Critical Care Research Network (CPCCRN) with Nationwide Children's Hospital as the primary site. The study design information currently displayed on this site refers to the dose-finding phase of the project.
Interventional
Phase 4
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Critical Injury (Trauma) in Children
Drug: GM-CSF
GM-CSF is to be administered IV at one of four possible dosing regimens (three days at a dose of 30, 62, or 125 mcg/m2 per day, or an extended dosing regimen of 125 mcg/m2/day through post-trauma 6) if severe innate immune suppression is identified on post-trauma days 1, 2, or 3.
Other Names:
  • sargramostim
  • leukine
Experimental: GM-CSF
GM-CSF is given in one of four treatment regimens (three days at a dose of 30, 62, or 125 mcg/m2/day, or extended dosing at 125 mcg/m2 through post-trauma day 6) to critically injured children who demonstrate severe reduction in innate immune function on post-trauma day 1, 2, or 3.
Intervention: Drug: GM-CSF
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
200
December 2018
June 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Admission to the PICU at a GIFT study site with a primary diagnosis of blunt or penetrating trauma that occurred within the last 72 hours.
  • Age 1 - 17 years
  • Provisional Injury Severity Score (ISS) > 10
  • Presence of an endotracheal tube at the time of enrollment

Exclusion Criteria:

  • DNR status or care team/family is considering plans for withdrawal of life-sustaining therapies.
  • Strong suspicion of injuries related to child abuse, in the opinion of the treating physician
  • Persistence (after treatment) of any of the following in the PICU before enrollment: Fixed, dilated pupils; Glasgow Coma Scale score of 3 (in the absence of neuromuscular blocking drugs); or presence of a new, severe neurologic injury at the time of enrollment which, in the opinion of the treating physician, is highly likely to lead to a diagnosis of brain death
  • Cardiopulmonary arrest requiring CPR documented by EMS or hospital personnel prior to subject identification
  • Burn injury of any kind (scald, fire, chemical)
  • Patients receiving acute or chronic immunosuppressive therapy (e.g., systemic corticosteroids, calcineurin inhibitors, mycophenolate, azathioprine) at the time of injury
  • Patients with severe leukopenia (white blood cell count < 1000 cells/mm3) at the time of injury as the result of myeloablative chemotherapy or radiation
  • Pregnancy
  • Autoimmune thrombocytopenia, myelodysplastic syndromes with > 20% marrow blast cells, or known allergy/hypersensitivity to GM-CSF
  • Previously enrolled in the GIFT study
Sexes Eligible for Study: All
1 Year to 17 Years   (Child)
No
Contact: Mark W Hall, MD 614-722-3438 Mark.Hall@NationwideChildrens.org
United States
 
 
NCT01495637
GIFT Study
R01GM094203 ( U.S. NIH Grant/Contract )
UG1HD083170 ( U.S. NIH Grant/Contract )
Yes
Not Provided
Plan to Share IPD: Yes
Plan Description: A public use data set will be made available on the website of the NICHD's Collaborative Pediatric Critical Care Research Network.
Supporting Materials: Study Protocol
Supporting Materials: Clinical Study Report (CSR)
Time Frame: Within one year of study completion.
Access Criteria: Access is through web request on the CPCCRN website
Mark Hall, Nationwide Children's Hospital
Mark Hall
  • National Institute of General Medical Sciences (NIGMS)
  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Principal Investigator: Mark W Hall, MD Nationwide Children's Hospital
Nationwide Children's Hospital
July 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP