GM-CSF for Multiple Organ Failure (GRACE-2 Trial)

Palo Alto (17 mi)

Age: < 18

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Nationwide Children's Hospital

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?The GRACE-2 study is a prospective, multi-center, double-blind, randomized controlled trial of the drug GM-CSF vs placebo in children with sepsis-induced multiple organ dysfunction syndrome (MODS) who have immunoparalysis with mild to moderate inflammation.

Eligibility Criteria

This trial is for children with sepsis-induced organ dysfunction who are between 40 weeks corrected gestational age and under 18 years old. They must have new organ issues due to sepsis, be in pediatric or cardiac intensive care, and not have certain conditions like allergies to GM-CSF, high risk of brain death, severe blood disorders, or a history of specific immune diseases.

Inclusion Criteria

I have or might have an infection that led to my multiple organ dysfunction syndrome.

I am older than 40 weeks corrected gestational age but younger than 18 years.

Exclusion Criteria

I have been diagnosed with HLH or MAS.

I am currently breastfeeding.

I have not taken anakinra or GM-CSF in the last 28 days.

My white blood cell count is low due to recent intense therapy.

I have a history of blood disorders like leukemia or low platelet counts due to an autoimmune condition.

Treatment Details

The GRACE-2 study tests if GM-CSF can help kids with sepsis-induced multiple organ failure whose immune systems are too weak. It's a blind test where neither doctors nor patients know who gets the real drug versus a placebo. Kids are chosen randomly to get one or the other.

2Treatment groups

Active Control

Placebo Group

Group I: GM-CSFActive Control1 Intervention

Intravenous GM-CSF 125 mcg/m2/day x 7 days

Group II: PlaceboPlacebo Group1 Intervention

Intravenous placebo x 7 days

GM-CSF is already approved in United States, European Union, Canada for the following indications:

🇺🇸 Approved in United States as Leukine for: