GM-CSF for Immunomodulation Following Trauma (GIFT) Study

NCT01495637 | Completed Phase 4 DMC

• 3 other identifiers: GIFT StudyR01GM094203UG1HD083170
• Study Type: interventional
• Target: 108
• Locations: 1 country
• Sites: 8

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.

Conditions

Critical Injury (Trauma) in Children

Keywords

pediatric; trauma; critical; GM-CSF; immune

Outcome Measures

Primary Outcomes (1)

• Immune function

  To identify the lowest immunostimulatory yet tolerable dose of GM-CSF that produces lasting improvement in innate immune function in treated children.

  7-days post-trauma

Secondary Outcomes (1)

• Nosocomial infection

  28-days post-trauma

Study Arms (1)

GM-CSF

EXPERIMENTAL

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

Interventions

GM-CSF DRUG

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.

Also known as: sargramostim, leukine

GM-CSF

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Mark Hall: lead
• National Institute of General Medical Sciences (NIGMS): collaborator
• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): collaborator

Study Sites (8)

Children's Hospital of Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48201, United States

Location

Washington University / St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

Cincinnati Children's Medical Center

Cincinnati, Ohio, 45229, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

MeSH Terms

Conditions

Wounds and Injuries

Interventions

Granulocyte-Macrophage Colony-Stimulating Factor; sargramostim

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Mark W Hall, MD

  Nationwide Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: December 13, 2011
• First Posted: December 20, 2011
• Study Start: December 1, 2011
• Primary Completion: February 1, 2022
• Study Completion: February 1, 2023
• Last Updated: September 13, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, CSR
• Time Frame: Within one year of study completion.
• Access Criteria: Access is through web request on the CPCCRN website

Locations

US (8)