Approaches and Decisions for Acute Pediatric TBI Trial

NCT ID: NCT04077411

Last Updated: 2023-12-08

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

1000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-02-20

Study Completion Date

2018-08-28

Brief Summary

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Approaches and Decisions in Acute Pediatric TBI Trial (ADAPT) is an international research study designed to evaluate the impact of interventions on the outcomes of children with severe traumatic brain injury.

Pediatric traumatic brain injury (TBI) is the leading killer of children, resulting in more than 7000 deaths and $2 billion in acute care costs each year. Despite this large burden of disease, advances in the field have been limited due to weak evidenced-based guidelines and the limitations of randomized, controlled trials (RCTs) to demonstrate efficacy of single treatment strategies due to wide treatment variability. ADAPT is a practical study design in a novel approach - an observational cohort study designed to evaluate the association of 6 aspects of pediatric TBI care with outcomes using statistical modeling to correct for confounding variables. Completion of this study will provide compelling evidence to change clinical practices, provide evidence for new Level II recommendations for future guidelines and lead to improved research protocols that would limit variability in TBI treatments - helping children immediately through better clinical practices and ultimately through more effective investigation.

Detailed Description

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Traumatic brain injury (TBI) is the leading cause of death in children in the US. According to the CDC, 7440 children died of TBI in 2005, but this likely underestimates the full burden of the disease. Based on the current best estimates for severe pediatric TBI (20% mortality, 50.6% unfavorable 6 mo outcome, mean age 9 y), each year 37,200 children suffer a severe TBI with up to 1.3 million life-years potentially adversely affected.

Incremental improvement in outcomes could make enormous differences for the health of children, but such advances have remained elusive. Dozens of injury mechanisms have been identified after experimental TBI, yet no mitigating treatments have been translated into clinical practice. Randomized controlled trials (RCTs) of therapies, from steroids to novel pharmaceutical agents to hypothermia, have failed for adult and pediatric TBI victims. Single-center experiences have contributed to understanding, yet these largely remain insufficiently powerful to change practice. Recently, evidenced-based guidelines for 15 aspects of pediatric TBI were published that provide no level I and only 4 Level II recommendations - with such recommendations indicating therapies that "must be done" or "should be considered" based on the literature, respectively. Disappointingly, 3 of these recommendations advised against specific interventions (hypothermia, steroids and immune-enhanced diets) - emphasizing the uncertainty of the effectiveness of many commonly used therapies that leads to wide variations in clinical practice. Unsurprisingly, significant variations of clinical outcomes and basic treatment strategies for TBI have been observed. The IMPACT study merged data from over 9000 adults with TBI from 11 trials and demonstrated significant variations in outcomes from clinical sites. Similar variations in outcomes in children with TBI can be found using various administrative databases, with mortality rates varying between 12.2% - 34.4% in 11 US states. There are also variations in strategies within an international consortium and a recently completed RCT - with marked variations in strategies for first-line intracranial hypertension treatments, prevention of common secondary insults and metabolic support after pediatric TBI.

The paucity of data to create robust guidelines, the failure of RCTs that tested a wide-variety of putative mechanisms and variations in outcomes and in clinical practices argues that the current understanding of contemporary therapies is inadequate. Because neither retrospective analyses from available databases nor self-reported variations in practices can determine optimal therapeutic strategies for these contemporaneous strategies, a new approach is urgently needed.

ADAPT is a large, prospective, observational cohort study using an international consortium including sites from the US, EU and UK. Children with severe TBI \[Glasgow coma scale (GCS) score ≤ 8 with intracranial pressure (ICP) monitoring, n = 1000, \>32 sites\] will be studied. The local standard of care at each site will be used and extensive data collection over the first 7 days after TBI will be performed to interrogate the effectiveness of strategies for intracranial hypertension, mitigation of specific secondary insults and metabolism.

Several statistical approaches, often used in comparative effectiveness research (CER) to control for measured confounding effects, will test the following aims:

Specific Aim 1: Compare the effectiveness of first-line intracranial hypertension strategies on outcome. Intracranial hypertension management is a mainstay of TBI care yet evidence for utilization the first-line therapies of cerebrospinal fluid (CSF) diversion and use of hyperosmolar solutions, is incomplete.

Aim 1a: Determine the effect of CSF diversion strategies (continuous drainage, intermittent drainage and none) on outcome. Aim 1b: Determine the effect of hyperosmolar therapies (hypertonic saline, mannitol) on outcome.

Specific Aim 2: Compare the effectiveness of strategies that mitigate specific secondary insults on outcome. Prophylactic hyperventilation (HV) and hypoxia may worsen outcome after TBI but have been inadequately studied.

Aim 2a: Determine the effect of prophylactic HV (CO2 \< 30 mm Hg) on outcome.

Aim 2b: Determine the effect of hypoxia detection with brain tissue oxygen monitoring (PbO2) on outcome.

Conditions

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Severe Traumatic Brain Injury

Keywords

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Severe Traumatic Brain Injury TBI Intracranial hypertension Cerebrospinal fluid Hypersmolar therapies Prophylactic hyperventilation Metabolism Neuropsychology

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Children with severe TBI

Children with severe Traumatic Brain Injury

Observational

Intervention Type OTHER

This is an observational study with no interventions.

Interventions

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Observational

This is an observational study with no interventions.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Admission to a study site for treatment of severe traumatic brain injury
* ICP monitor placed as part of the child's standard care
* Glasgow Coma Scale (GCS) ≤ 8 after resuscitation
* Age 0 - 18 y

Exclusion Criteria

* 1\. ICP Monitor placed at another hospital
* 2\. Diagnosis of pregnancy in clinical subject
Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role collaborator

University of Pittsburgh

OTHER

Sponsor Role lead

Responsible Party

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Stephen Wisniewski

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Michael J Bell, MD

Role: PRINCIPAL_INVESTIGATOR

University of Pittsburgh Medical Center

Stephen R Wisniewski, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Pittsburgh

Locations

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Children's Hospital of Alabama

Birmingham, Alabama, United States

Site Status

Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

University of California, San Diego / Rady's Children's Hospital

La Jolla, California, United States

Site Status

Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status

UCLA Mattel Children's Hospital

Los Angeles, California, United States

Site Status

UC Davis Medical Center

West Sacramento, California, United States

Site Status

Children's Hospital Colorado

Denver, Colorado, United States

Site Status

Children's National Medical Center

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

Site Status

Miami Children's Hospital

Miami, Florida, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

University of Iowa Children's Hospita

Iowa City, Iowa, United States

Site Status

John Hopkins Children's Center of Baltimore

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Boston Children's Hospital / Harvard University

Boston, Massachusetts, United States

Site Status

Wayne State University in Detroit / Children's Hospital of Michigan

Detroit, Michigan, United States

Site Status

Washington University - St. Louis

St Louis, Missouri, United States

Site Status

University of Nebraska Medical Center

Omaha, Nebraska, United States

Site Status

Carolinas Medical Center Levine Children's Hospital

Charlotte, North Carolina, United States

Site Status

University of Cincinnati / Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Penn State University - Hershey

Hershey, Pennsylvania, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

University of Tennessee / Le Bonheur Children's Hospita

Memphis, Tennessee, United States

Site Status

UT Southwestern / Children's Medical Center at Dallas

Dallas, Texas, United States

Site Status

Texas Children's Hospital (Baylor College of Medicine)

Houston, Texas, United States

Site Status

Primary Children's Hospital

Salt Lake City, Utah, United States

Site Status

Children's Hospital of Richmond at VCU

Richmond, Virginia, United States

Site Status

University of Washington - Seattle

Seattle, Washington, United States

Site Status

University of Wisconsin - Madison

Madison, Wisconsin, United States

Site Status

Children's Health Queensland Hospital and Health Service

Brisbane, , Australia

Site Status

Royal Children's Hospital

Melbourne, , Australia

Site Status

Princess Margaret Hospital

Perth, , Australia

Site Status

All India Institute of Medical Sciences

New Delhi, , India

Site Status

Erasmus Medical Center Children's Hospital

Rotterdam, , Netherlands

Site Status

Starship Children's Hospital

Auckland, , New Zealand

Site Status

University of Cape Town

Cape Town, , South Africa

Site Status

Hospital Vall d'Hebron

Barcelona, , Spain

Site Status

Birmingham Children's Hospita

Birmingham, , United Kingdom

Site Status

University Hospitals Bristol

Bristol, , United Kingdom

Site Status

Addenbrookes Hospital

Cambridge, , United Kingdom

Site Status

Leeds Teaching Hospital

Leeds, , United Kingdom

Site Status

Alder Hey Children's

Liverpool, , United Kingdom

Site Status

King's College Hospital

London, , United Kingdom

Site Status

Great Ormond Street

London, , United Kingdom

Site Status

Newcastle upon Tyne Hospital

Newcastle upon Tyne, , United Kingdom

Site Status

University Hospital Southampton

Southampton, , United Kingdom

Site Status

Royal Manchester Children's Hospital

Victoria, , United Kingdom

Site Status

Countries

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United States Australia India Netherlands New Zealand South Africa Spain United Kingdom

References

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Sarnaik A, Ferguson NM, O'Meara AMI, Agrawal S, Deep A, Buttram S, Bell MJ, Wisniewski SR, Luther JF, Hartman AL, Vavilala MS; Investigators of the ADAPT Trial. Age and Mortality in Pediatric Severe Traumatic Brain Injury: Results from an International Study. Neurocrit Care. 2018 Jun;28(3):302-313. doi: 10.1007/s12028-017-0480-x.

Reference Type BACKGROUND
PMID: 29476389 (View on PubMed)

Bell MJ, Adelson PD, Wisniewski SR; Investigators of the ADAPT Study,. Challenges and opportunities for pediatric severe TBI-review of the evidence and exploring a way forward. Childs Nerv Syst. 2017 Oct;33(10):1663-1667. doi: 10.1007/s00381-017-3530-y. Epub 2017 Sep 6.

Reference Type BACKGROUND
PMID: 29149394 (View on PubMed)

Miller Ferguson N, Sarnaik A, Miles D, Shafi N, Peters MJ, Truemper E, Vavilala MS, Bell MJ, Wisniewski SR, Luther JF, Hartman AL, Kochanek PM; Investigators of the Approaches and Decisions in Acute Pediatric Traumatic Brain Injury (ADAPT) Trial. Abusive Head Trauma and Mortality-An Analysis From an International Comparative Effectiveness Study of Children With Severe Traumatic Brain Injury. Crit Care Med. 2017 Aug;45(8):1398-1407. doi: 10.1097/CCM.0000000000002378.

Reference Type BACKGROUND
PMID: 28430697 (View on PubMed)

Larsen GY, Schober M, Fabio A, Wisniewski SR, Grant MJ, Shafi N, Bennett TD, Hirtz D, Bell MJ. Structure, Process, and Culture Differences of Pediatric Trauma Centers Participating in an International Comparative Effectiveness Study of Children with Severe Traumatic Brain Injury. Neurocrit Care. 2016 Jun;24(3):353-60. doi: 10.1007/s12028-015-0218-6.

Reference Type BACKGROUND
PMID: 26627225 (View on PubMed)

Ahmed N, Russo L, Kuo YH. Levetiracetam or Phenytoin as Prophylaxis for Status Epilepticus: Secondary Analysis of the "Approaches and Decisions in Acute Pediatric Traumatic Brain Injury Trial" (ADAPT) Dataset, 2014-2017. Pediatr Crit Care Med. 2024 Aug 1;25(8):710-719. doi: 10.1097/PCC.0000000000003526. Epub 2024 May 8.

Reference Type DERIVED
PMID: 38717237 (View on PubMed)

Surtees TL, Kumar I, Garton HJL, Rivas-Rodriguez F, Parmar H, McCaffery H, Riebe-Rodgers J, Shellhaas RA. Levetiracetam Prophylaxis for Children Admitted With Traumatic Brain Injury. Pediatr Neurol. 2022 Jan;126:114-119. doi: 10.1016/j.pediatrneurol.2021.10.009. Epub 2021 Oct 19.

Reference Type DERIVED
PMID: 34839268 (View on PubMed)

Other Identifiers

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U01NS081041

Identifier Type: NIH

Identifier Source: secondary_id

View Link

PRO13020047

Identifier Type: -

Identifier Source: org_study_id