GM-CSF for Multiple Organ Failure

(GRACE-2 Trial)

Recruiting at25 trial locations

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.

Research Team

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.

Admission to the PICU or CICU

Onset of ≥ 2 new organ dysfunctions within the last 3 calendar days (compared to pre-sepsis baseline) as measured by the modified Proulx criteria

Exclusion Criteria

Moribund condition in which the patient is unlikely to survive the next 48 hours in opinion of the clinical team

Weight <3kg

Previous enrollment in the GRACE-2 study

Treatment Details

Interventions

• GM-CSF (Cytokine)
• Placebo (Other)

Trial Overview 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.

Participant Groups

Group I: GM-CSF Active Control 1 Intervention

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

Group II: Placebo Placebo Group 1 Intervention

Intravenous placebo x 7 days

GM-CSF is already approved in Canada for the following indications:

Approved in Canada as Leukine for:

• Neutropenia
• Bone Marrow Transplantation
• Leukemia
• Lymphoma

Who Is Running the Clinical Trial?

Nationwide Children's Hospital

Lead Sponsor

Trials

354

Recruited

5,228,000+

Catherine Krawczeski

Nationwide Children's Hospital

Chief Medical Officer

MD

Timothy C. Robinson

Nationwide Children's Hospital

Chief Executive Officer since 2019

BSc in Psychology and Business Administration from Indiana University

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborator

Trials

2,103

Recruited

2,760,000+

Dr. Diana W. Bianchi

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Chief Executive Officer since 2016

MD from Stanford University

Dr. Alison Cernich

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Chief Medical Officer since 2020

PhD in Clinical Psychology from University of Maryland