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Metabolic and Hemodynamic Reserve in Pediatric SCA

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ClinicalTrials.gov Identifier: NCT04406818
Recruitment Status : Recruiting
First Posted : May 28, 2020
Last Update Posted : March 29, 2022
Sponsor:
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
Washington University School of Medicine

Tracking Information
First Submitted Date  ICMJE May 22, 2020
First Posted Date  ICMJE May 28, 2020
Last Update Posted Date March 29, 2022
Actual Study Start Date  ICMJE June 15, 2021
Estimated Primary Completion Date December 31, 2025   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 27, 2020)
Gray Matter cortical thickness [ Time Frame: 3 years ]
Mean whole brain cortical thickness on high resolution T1 images
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 1, 2021)
  • Total Brain volume [ Time Frame: 3 years ]
    Total brain volume (gray matter and white matter) on high resolution T1 image
  • Cerebrovascular Reactivity [ Time Frame: 15 minutes ]
    Change in blood flow as measured by MRI in response to carbon dioxide
Original Secondary Outcome Measures  ICMJE
 (submitted: May 27, 2020)
Total Brain volume [ Time Frame: 3 years ]
Total brain volume (gray matter and white matter) on high resolution T1 image
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Metabolic and Hemodynamic Reserve in Pediatric SCA
Official Title  ICMJE The Role of Metabolic and Hemodynamic Reserve in Age-Related Brain Vulnerability in Pediatric Sickle Cell Anemia
Brief Summary

The purpose of this research study is to better understand how blood flow and metabolism change can influence brain development in the early decades of life. SCA participants and healthy controls are age and sex-matched for comparison. Within the SCA cohort, children with infarcts may have thinner cortices than those without, reflecting a greater loss.

The investigators will examine brain blood flow and metabolism using magnetic resonance imaging (MRI). The brain's blood vessels expand and constrict to regulate blood flow based on the brain's needs. The amount of expanding and contracting the blood vessels may vary by age. The brain's blood flow changes in small ways during everyday activities, such exercise, deep concentration, or normal brain growth. Significant illness or psychological stress may increase the brain's metabolic demand or cause other bigger changes in blood flow. If blood vessels are not able to expand to give more blood flow when metabolic demand is high, the brain may not get all of the oxygen it needs. In extreme circumstances, if the brain is unable to get enough oxygen for a long time, a stroke may occur. Sometimes small strokes occur without other noticeable changes and are only detectable on an MRI. These are sometimes called "silent strokes." In less extreme circumstances, not having a full oxygen supply may cause the brain to grow and develop more slowly than when it has a full supply.

One way to test the ability of blood vessels to expand is by measuring blood flow while breathing in carbon dioxide. Carbon dioxide causes blood vessels in the brain to dilate without increasing brain metabolism.

During this study participants may be asked to undergo a blood draw, MRI, cognitive assessments, and brief questionnaires. The study team will use a special mask to control the amount of carbon dioxide the participants breathe in.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Condition  ICMJE
  • Child, Only
  • Brain Diseases
  • Sickle Cell Disease
  • Anemia, Sickle Cell
Intervention  ICMJE Drug: Carbon Dioxide
Participants inhale carbon dioxide while in magnetic resonance imaging scan to measure cerebrovascular reactivity
Study Arms  ICMJE
  • Active Comparator: Healthy Control
    Intervention: Drug: Carbon Dioxide
  • Active Comparator: Sickle Cell Anemia
    Intervention: Drug: Carbon Dioxide
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: July 1, 2021)
120
Original Estimated Enrollment  ICMJE
 (submitted: May 27, 2020)
250
Estimated Study Completion Date  ICMJE March 31, 2026
Estimated Primary Completion Date December 31, 2025   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Healthy Controls:

  • Healthy controls ages 4-21 years of age
  • Able to participate in MRI scan without sedation
  • Not currently pregnant
  • No significant psychiatric history, defined as having a severe psychiatric diagnosis, per PI discretion
  • No history of epilepsy
  • No history of stroke or cerebrovascular disease
  • May have occasional headaches if not taking a daily preventative medication for headaches
  • Not on vasodilatory medication, such as sildenafil or verapamil

Sickle Cell Anemia Participants:

  • Ages 4-21 years of age
  • Hb SS or SBeta-thal
  • Able to participate in MRI scan without sedation
  • Not currently pregnant
  • Not on vasodilatory medication, such as sildenafil or verapamil
  • No known vasculopathy
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE
Contact: Kristin P Guilliams, MD 3144546120 kristinguilliams@wustl.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04406818
Other Study ID Numbers  ICMJE 771237
R01NS121065 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: The proposed research assets will include MRI scan data and CO2 breathing data from 120 subjects, including pediatric subjects. As children are a vulnerable population, the project team will make the data (with any personally identifiable information redacted) available to qualified investigators under a data sharing agreement that provides for (1) a commitment to using for research purposes only, (2) a commitment to securing the data using appropriate computer technology, and (3) a commitment to destroying the data after analyses are completed. This will be available upon request within 6 months of the grant period ending.
Supporting Materials: Informed Consent Form (ICF)
Time Frame: At study conclusion
Access Criteria: Upon request by qualified researchers
Current Responsible Party Washington University School of Medicine
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Washington University School of Medicine
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE National Institute of Neurological Disorders and Stroke (NINDS)
Investigators  ICMJE
Principal Investigator: Kristin P Guilliams, MD Washington University School of Medicine
PRS Account Washington University School of Medicine
Verification Date March 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP