NCT04113434

Brief Summary

The overall goal of the study is to risk stratify pediatric Acute Respiratory Distress Syndrome (ARDS) patients and to identify sub-phenotypes with shared biology in order to appropriately target therapies in future trials. This is a prospective, multicenter study of 500 intubated children with ARDS, with planned blood collection within 24 hours of ARDS onset and subsequent measurement of plasma protein biomarkers and peripheral blood gene expression.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
513

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jan 2020

Longer than P75 for all trials

Geographic Reach
1 country

16 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

October 1, 2019

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 2, 2019

Completed
3 months until next milestone

Study Start

First participant enrolled

January 7, 2020

Completed
5.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 15, 2025

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2025

Completed
1.1 years until next milestone

Results Posted

Study results publicly available

April 20, 2026

Completed
Last Updated

April 20, 2026

Status Verified

April 1, 2026

Enrollment Period

5.1 years

First QC Date

October 1, 2019

Results QC Date

February 9, 2026

Last Update Submit

April 9, 2026

Conditions

Keywords

Acute Respiratory Distress SyndromeARDS

Outcome Measures

Primary Outcomes (3)

  • 28 Day Mortality in Pediatric ARDS.

    28 day all cause mortality.

    28 days

  • Presence of Two or More Endotypes in Pediatric ARDS.

    Stratify pediatric ARDS into sub-phenotypes using a known 100-gene expression-based classifier to group subjects according to shared underlying biology.

    Within 24 hours of ARDS onset

  • Occurrence of de Novo Sub-phenotypes in Pediatric ARDS Using Biomarkers and Whole Genome Transcriptomics of Peripheral Blood.

    Occurrence of de novo sub-phenotypes in pediatric ARDS using 12 protein biomarkers and whole genome transcriptomics of peripheral blood.

    Within 24 hours of ARDS onset.

Secondary Outcomes (1)

  • Ventilator-free Days at 28 Days.

    28 days

Eligibility Criteria

Age44 Weeks - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodProbability Sample
Study Population

Infants to adolescents between the ages of 44 weeks and 17.5 years, who are admitted to PICU for acute respiratory failure requiring invasive mechanical ventilation.

You may qualify if:

  • acute (≤ 7 days of risk factor) respiratory failure requiring invasive mechanical ventilation
  • age \> 44 weeks corrected gestational age and \< 17.5 years
  • invasive mechanical ventilation via endotracheal tube
  • bilateral infiltrates on chest radiograph
  • oxygenation index (OI) ≥ 4; or oxygen saturation index (OSI) ≥ 5 on 2 consecutive measurements at least 4 hours apart but \< 24 hours apart
  • invasively ventilated ≤ 7 days before meeting above radiographic and oxygenation criteria

You may not qualify if:

  • weight \< 3 kilograms
  • cyanotic congenital heart disease (other than Patent Foramen Ovale (PFO) or Patent Ductus Arteriosus (PDA))
  • tracheostomy at time of screening
  • invasively ventilated for \> 7 days when meet ARDS criteria above
  • cardiac failure as predominant cause of respiratory failure
  • primary obstructive airway disease (asthma, bronchiolitis) by judgement of clinician as the primary cause of respiratory failure
  • alternative known chronic lung disease as cause of respiratory failure (cystic fibrosis, eosinophilic pneumonia, interstitial pneumonitis, pulmonary hemosiderosis, cryptogenic organizing pneumonia)
  • severe neurologic morbidity not expected to survive \> 72 hours
  • any limitations of care at time of screening
  • previous enrollment in this study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (16)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Variety Children's Hospital D/B/A Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Children's Healthcare of Atlanta - Emory

Atlanta, Georgia, 30322, United States

Location

Riley Children's at Indiana University Health

Indianapolis, Indiana, 46202, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Washington University

St Louis, Missouri, 63110, United States

Location

Cooperman Barnabas Medical Center

Livingston, New Jersey, 07039, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Akron Children's Hospital

Akron, Ohio, 44308, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Penn State Hershey Children's Hospital

Hershey, Pennsylvania, 17033, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Texas Children's Hospital / Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (1)

  • Whitney JE, Johnson GM, Varisco BM, Raby BA, Yehya N. Biomarker-Based Risk Stratification Tool in Pediatric Acute Respiratory Distress Syndrome: Single-Center, Longitudinal Validation in a 2014-2019 Cohort. Pediatr Crit Care Med. 2024 Jul 1;25(7):599-608. doi: 10.1097/PCC.0000000000003512. Epub 2024 Apr 9.

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma

MeSH Terms

Conditions

Respiratory Distress Syndrome

Condition Hierarchy (Ancestors)

Lung DiseasesRespiratory Tract DiseasesRespiration Disorders

Results Point of Contact

Title
Dr. Nadir Yehya
Organization
Children's Hospital of Philadelphia

Study Officials

  • Nadir Yehya, M.D.

    Children's Hospital of Philadelphia

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 1, 2019

First Posted

October 2, 2019

Study Start

January 7, 2020

Primary Completion

February 15, 2025

Study Completion

April 1, 2025

Last Updated

April 20, 2026

Results First Posted

April 20, 2026

Record last verified: 2026-04

Data Sharing

IPD Sharing
Will share

Specific consent will be obtained to store blood and use data for future research. We will share data with the research community using the Biologic Specimen and Data Repository Information Coordinating Center (BioLINCC) program at NHLBI, and we will follow suggested guidelines for de-identifying the data (coding of site IDs, conversion of dates to study days). Pursuant to the NIH policy, all data obtained from this proposal will be made available for research by qualified individuals within the scientific community after publication of the proposed studies. Gene expression data from Aims 2 and 3 will be made available simultaneous with publication of the results of these aims by uploading to the Gene Expression Omnibus. For dissemination of plasma biomarker results and the associated clinical dataset, we will use BioLINCC program at NHLBI, with release of a de-identified dataset with untraceable identifiers within 2 years of publication of the main manuscript.

Shared Documents
STUDY PROTOCOL, ICF
Time Frame
Gene expression data will be released to Gene Expression Omnibus simultaneously with the publication of the data. Biomarker data will be released within 2 years of publication of the main manuscript.
Access Criteria
We will follow NHLBI's guidelines for accessing BioLINCC data.

Locations