Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2016 by Nationwide Children's Hospital
Sponsor:
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
ClinicalTrials.gov Identifier:
NCT01495637
First received: December 13, 2011
Last updated: May 4, 2016
Last verified: May 2016

December 13, 2011
May 4, 2016
December 2011
June 2017   (final data collection date for primary outcome measure)
Immune function [ Time Frame: 7-days post-trauma ] [ Designated as safety issue: Yes ]
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 ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT01495637 on ClinicalTrials.gov Archive Site
Nosocomial infection [ Time Frame: 28-days post-trauma ] [ Designated as safety issue: Yes ]
  • Innate immune function [ Time Frame: Day 4 post-trauma ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]
  • Innate Immune Function [ Time Frame: Day 14 post-trauma ] [ Designated as safety issue: No ]
    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
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Critical Injury (Trauma) in Children
Drug: GM-CSF
GM-CSF is to be administered IV for three days at a dose of 30, 62, or 125 mcg/m2 per day 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 for three days at a dose of 30, 62, or 125 mcg/m2/day 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
June 2017
June 2017   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Admission to the PICU at a CPCCRN 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
Both
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
Yes
Yes
Not Provided
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
May 2016

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