NCT05266001

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
141

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Jun 2022

Typical duration for phase_3

Geographic Reach
1 country

25 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 23, 2022

Completed
9 days until next milestone

First Posted

Study publicly available on registry

March 4, 2022

Completed
3 months until next milestone

Study Start

First participant enrolled

June 14, 2022

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 10, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 10, 2025

Completed
Last Updated

April 9, 2026

Status Verified

April 1, 2026

Enrollment Period

3.2 years

First QC Date

February 23, 2022

Last Update Submit

April 8, 2026

Conditions

Outcome Measures

Primary Outcomes (1)

  • Cumulative 28-day Pediatric Logistic Organ Dysfunction (PELOD)-2 score

    Cumulative Pediatric Logistic Organ Dysfunction (PELOD)-2 score through 28 days post-randomization. The range of PELOD-2 score for a given day is 0 - 33 with a higher number being worse.

    28 days from randomization

Secondary Outcomes (2)

  • 3-month health-related quality of life

    3 months post-randomization

  • 3-month functional status

    3 months post-randomization

Study Arms (2)

GM-CSF

ACTIVE COMPARATOR

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

Drug: GM-CSF

Placebo

PLACEBO COMPARATOR

Intravenous placebo x 7 days

Other: Placebo

Interventions

GM-CSFDRUG

same as arm/group description

GM-CSF
PlaceboOTHER

same as arm/group description

Placebo

Eligibility Criteria

Age1 Day - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • ≥ 40 weeks corrected gestational age to \< 18 years; AND
  • Admission to the PICU or CICU; AND
  • Onset of ≥ 2 new organ dysfunctions within the last 3 calendar days (compared to pre-sepsis baseline) as measured by the modified Proulx criteria; AND
  • Documented or suspected infection as the MODS inciting event.

You may not qualify if:

  • Weight \<3kg; OR
  • Limitation of care order at the time of screening; OR
  • Patients at high likelihood of progression to brain death in opinion of the clinical team; OR
  • Moribund condition in which the patient is unlikely to survive the next 48 hours in opinion of the clinical team; OR
  • History of myeloid leukemia, myelodysplasia, or autoimmune thrombocytopenia; OR
  • Current or prior diagnosis of hemophagocytic lymphohistiocytosis or macrophage activation syndrome; OR
  • Peripheral white blood cell count \< 1,000 cells/mm3 as the result of myeloablative therapy OR receipt of myeloablative therapy within the previous 14 days; OR
  • Known allergy to GM-CSF; OR
  • Known pregnancy; OR
  • Lactating females; OR
  • Receipt of anakinra or GM-CSF within the previous 28 days; OR
  • Resolution of MODS by MODS Day 2; OR
  • Previous enrollment in the GRACE-2 study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (25)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

Benioff Children's Hospital - Oakland

Oakland, California, 64609, United States

Location

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

University of California - Davis

Sacramento, California, 95618, United States

Location

Benioff Children's Hospital - Mission Bay

San Francisco, California, 94143, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center

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

Location

CS Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

Location

Children's Hospital of Michigan

Detroit, Michigan, 48202, United States

Location

Children's Hospital of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Mercy Children's Hospital

Kansas City, Missouri, 64108, United States

Location

Duke University

Durham, North Carolina, 27705, United States

Location

Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Pennsylvania State University

Hershey, Pennsylvania, 17033, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15260, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Children's Hospital of San Antonio

San Antonio, Texas, 78207, United States

Location

Primary Children's Hospital

Salt Lake City, Utah, 84158, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53233, United States

Location

Related Publications (1)

  • VanBuren JM, Hall M, Zuppa AF, Mourani PM, Carcillo J, Dean JM, Watt K, Holubkov R. The Design of Nested Adaptive Clinical Trials of Multiple Organ Dysfunction Syndrome Children in a Single Study. Pediatr Crit Care Med. 2023 Dec 1;24(12):e635-e646. doi: 10.1097/PCC.0000000000003332. Epub 2023 Jul 27.

MeSH Terms

Interventions

Granulocyte-Macrophage Colony-Stimulating Factor

Intervention Hierarchy (Ancestors)

Colony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: Prospective, randomized, double-blind, placebo-controlled clinical trial of the drug GM-CSF vs placebo
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Chief, Critical Care Medicine

Study Record Dates

First Submitted

February 23, 2022

First Posted

March 4, 2022

Study Start

June 14, 2022

Primary Completion

September 10, 2025

Study Completion

September 10, 2025

Last Updated

April 9, 2026

Record last verified: 2026-04

Locations