GM-CSF for Immunomodulation Following Trauma (GIFT) Study

• Status: Active, not recruiting
• Trial end date: 2021-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Mark Hall
• Collaborators: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); National Institute of General Medical Sciences (NIGMS)
• Treatments: Sargramostim

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