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Metabolic and Hemodynamic Reserve in Pediatric SCA

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04406818
Recruitment Status : Recruiting
First Posted : May 28, 2020
Last Update Posted : February 21, 2023
Sponsor:
Collaborator:
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
Washington University School of Medicine

Study Description
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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.


Condition or disease Intervention/treatment Phase
Child, Only Brain Diseases Sickle Cell Disease Anemia, Sickle Cell Drug: Carbon Dioxide Not Applicable

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: The Role of Metabolic and Hemodynamic Reserve in Age-Related Brain Vulnerability in Pediatric Sickle Cell Anemia
Actual Study Start Date : June 30, 2021
Estimated Primary Completion Date : March 31, 2026
Estimated Study Completion Date : March 31, 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia

Arms and Interventions
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Arm Intervention/treatment
Active Comparator: Healthy Control Drug: Carbon Dioxide
Participants inhale carbon dioxide while in magnetic resonance imaging scan to measure cerebrovascular reactivity
Active Comparator: Sickle Cell Anemia Drug: Carbon Dioxide
Participants inhale carbon dioxide while in magnetic resonance imaging scan to measure cerebrovascular reactivity


Outcome Measures
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Primary Outcome Measures :
  1. Gray Matter cortical thickness [ Time Frame: 3 years ]
    Mean whole brain cortical thickness on high resolution T1 images


Secondary Outcome Measures :
  1. Total Brain volume [ Time Frame: 3 years ]
    Total brain volume (gray matter and white matter) on high resolution T1 image

  2. Cerebrovascular Reactivity [ Time Frame: 15 minutes ]
    Change in blood flow as measured by MRI in response to carbon dioxide


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04406818


Contacts
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Contact: Kristin P Guilliams, MD 3144546120 kristinguilliams@wustl.edu

Locations
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United States, Missouri
Washington University in St. Louis Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Kristin P Guillliams, MD    314-454-6120    kristinguilliams@wustl.edu   
Sponsors and Collaborators
Washington University School of Medicine
National Institute of Neurological Disorders and Stroke (NINDS)
Investigators
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Principal Investigator: Kristin P Guilliams, MD Washington University School of Medicine
More Information
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Responsible Party: Washington University School of Medicine
ClinicalTrials.gov Identifier: NCT04406818    
Other Study ID Numbers: 771237
R01NS121065 ( U.S. NIH Grant/Contract )
First Posted: May 28, 2020    Key Record Dates
Last Update Posted: February 21, 2023
Last Verified: February 2023
Individual Participant Data (IPD) Sharing Statement:
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

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Washington University School of Medicine:
sickle cell anemia
cerebrovascular reactivity
cerebral oxygen metabolism
cortical thickness
 gray matter
carbon dioxide
magnetic resonance imaging
brain development
Additional relevant MeSH terms:
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Brain Diseases
Anemia
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
 Anemia, Hemolytic
Hemoglobinopathies
Genetic Diseases, Inborn
Central Nervous System Diseases
Nervous System Diseases