Linking Endotypes and Outcomes in Pediatric Acute Respiratory Distress Syndrome

NCT ID: NCT04113434

Last Updated: 2025-04-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

500 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-01-07

Study Completion Date

2025-04-01

Brief Summary

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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.

Detailed Description

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Investigators will measure pre-determined biomarkers with known or suspected association with ARDS severity or outcome. Simultaneously, investigators will measure gene expression of peripheral blood. Both plasma biomarkers and gene expression profiles will be analyzed using various machine learning techniques, including classification and regression tree, latent class analysis, and hierarchical clustering with the goal of identifying sub-phenotypes of ARDS. These sub-phenotypes will be examined for association with outcome (primary is 28-day mortality), and explicitly tested for variation in response to exogenous treatments (e.g., corticosteroids).

Conditions

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Acute Respiratory Distress Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

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

Exclusion Criteria

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

44 Weeks

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Akron Children's Hospital

OTHER

Sponsor Role collaborator

Children's Hospital and Health System Foundation, Wisconsin

OTHER

Sponsor Role collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role collaborator

Children's Mercy Hospital Kansas City

OTHER

Sponsor Role collaborator

Milton S. Hershey Medical Center

OTHER

Sponsor Role collaborator

Nationwide Children's Hospital

OTHER

Sponsor Role collaborator

Nicklaus Children's Hospital

UNKNOWN

Sponsor Role collaborator

Indiana University

OTHER

Sponsor Role collaborator

Cooperman Barnabas Medical Center

UNKNOWN

Sponsor Role collaborator

Baylor College of Medicine

OTHER

Sponsor Role collaborator

Columbia University

OTHER

Sponsor Role collaborator

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role collaborator

Arkansas Children's Hospital Research Institute

OTHER

Sponsor Role collaborator

Children's Healthcare of Atlanta

OTHER

Sponsor Role collaborator

Children's Hospital Colorado

OTHER

Sponsor Role collaborator

Washington University School of Medicine

OTHER

Sponsor Role collaborator

Children's Hospital of Philadelphia

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Nadir Yehya, M.D.

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Locations

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Arkansas Children's Hospital

Little Rock, Arkansas, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

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

Miami, Florida, United States

Site Status

Children's Healthcare of Atlanta - Emory

Atlanta, Georgia, United States

Site Status

Riley Children's at Indiana University Health

Indianapolis, Indiana, United States

Site Status

Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Washington University

St Louis, Missouri, United States

Site Status

Cooperman Barnabas Medical Center

Livingston, New Jersey, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

Akron Children's Hospital

Akron, Ohio, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Penn State Hershey Children's Hospital

Hershey, Pennsylvania, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Texas Children's Hospital / Baylor College of Medicine

Houston, Texas, United States

Site Status

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

References

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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.

Reference Type DERIVED
PMID: 38591949 (View on PubMed)

Other Identifiers

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R01HL148054

Identifier Type: NIH

Identifier Source: secondary_id

View Link

19-016271

Identifier Type: -

Identifier Source: org_study_id

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