Child, OnlyNCT04406818
Metabolic and Hemodynamic Reserve in Pediatric SCA
A study testing Carbon Dioxide in people with Child, Only and Brain Diseases, run by Washington University School of Medicine at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 4 to 21 yearsHealthy volunteers can take part
- Conditions the study is for
- Child, Only, Brain Diseases, Sickle Cell Disease
- What is being tested?
- Carbon Dioxide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jun 2021to 31 Mar 2027
- Phase
- Interventional
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 21 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
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
Trial locations and contacts
1 site in 1 country.
United States1 site
Washington University in St. Louis
St Louis, Missouri, 63110
About this study
Official title: The Role of Metabolic and Hemodynamic Reserve in Age-Related Brain Vulnerability in Pediatric Sickle Cell Anemia
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.
Read more
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.
- Conditions
- Child, Only, Brain Diseases, Sickle Cell Disease, Anemia, Sickle Cell
- Being tested
- Carbon Dioxide
- Keywords
- sickle cell anemia, cerebrovascular reactivity, cerebral oxygen metabolism, cortical thickness, gray matter, carbon dioxide, magnetic resonance imaging, brain development
- Medical subject headings
- Brain Diseases, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Carbon DioxideDrug
Participants inhale carbon dioxide while in magnetic resonance imaging scan to measure cerebrovascular reactivity
| Study group | Role | What they receive |
|---|---|---|
| Healthy Control | Active comparator | Not described Receives: Drug: Carbon Dioxide |
| Sickle Cell Anemia | Active comparator | Not described Receives: Drug: Carbon Dioxide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Gray Matter cortical thickness
Mean whole brain cortical thickness on high resolution T1 images
Measured: 3 years
Secondary outcomes (2)
Total Brain volume
Total brain volume (gray matter and white matter) on high resolution T1 image
Measured: 3 years
Cerebrovascular Reactivity
Change in blood flow as measured by MRI in response to carbon dioxide
Measured: 15 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2020
- Study start30 Jun 2021
- Primary completion31 Mar 2027 (estimated)
- Study completion31 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Child, OnlyStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Sponsor
- Investigators
- Kristin P Guilliams, MD, Principal investigator, Washington University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04406818
- Sponsor's protocol ID
- 202106016
- Other IDs
- R01NS121065
- Status last verified
- Aug 2026
- First submitted
- 22 May 2020
- Last updated
- 19 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.