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

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01495637
Recruitment Status : Active, not recruiting
First Posted : December 20, 2011
Last Update Posted : July 15, 2020
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

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Critical Injury (Trauma) in Children Drug: GM-CSF Phase 4

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 200 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: GM-CSF for Immunomodulation Following Trauma (GIFT) Study
Actual Study Start Date : December 2011
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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.
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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Nosocomial infection [ Time Frame: 28-days post-trauma ]
    The development of hospital-acquired infection through post-trauma day 28



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   1 Year to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01495637


Locations
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United States, Colorado
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
United States, Michigan
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
United States, Missouri
Washington University / St. Louis Children's Hospital
Saint Louis, Missouri, United States, 63110
United States, Ohio
Cincinnati Children's Medical Center
Cincinnati, Ohio, United States, 45229
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Sponsors and Collaborators
Mark Hall
National Institute of General Medical Sciences (NIGMS)
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Investigators
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Principal Investigator: Mark W Hall, MD Nationwide Children's Hospital
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Responsible Party: Mark Hall, Professor of Pediatrics, Nationwide Children's Hospital
ClinicalTrials.gov Identifier: NCT01495637    
Other Study ID Numbers: GIFT Study
R01GM094203 ( U.S. NIH Grant/Contract )
UG1HD083170 ( U.S. NIH Grant/Contract )
First Posted: December 20, 2011    Key Record Dates
Last Update Posted: July 15, 2020
Last Verified: July 2020
Individual Participant Data (IPD) Sharing Statement:
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
Clinical Study Report (CSR)
Time Frame: Within one year of study completion.
Access Criteria: Access is through web request on the CPCCRN website
Keywords provided by Mark Hall, Nationwide Children's Hospital:
pediatric
trauma
critical
GM-CSF
immune
Additional relevant MeSH terms:
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Wounds and Injuries
Sargramostim
Immunologic Factors
Physiological Effects of Drugs