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NCT Number: NCT06477289

Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease

This study will examine sleep disordered breathing and sleep quality in participants (ages 12-25) diagnosed with sickle cell disease of any genotype. We will utilize remote peripheral arterial tonometry (PAT) and questionnaires to evaluate difficulties with sleep. PAT assessments will occur remotely in the homes of participants.

Neurocognitive, behavioral, and neuroimaging evaluations will occur on the same day as a routine clinic visit.

Primary Objective:

Evaluate the relationship between nocturnal oxyhemoglobin saturation (SpO2) and neurocognitive functioning (working memory and verbal comprehension) in individuals (ages 12-25) diagnosed with sickle cell disease controlling for age, genotype, and social vulnerability.

Secondary Objective:

Assess differences in white matter integrity, silent cerebral infarcts, neuroinflammation, and functional connectivity among individuals (ages 12-25) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age.

Assess differences in self- and caregiver-reported mood and pain severity among individuals (ages 12-25) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age.

Exploratory Objectives:

Explore the relationship between nocturnal oxyhemoglobin saturation (SpO2) and neurocognitive functioning (attention, processing speed, verbal memory, visual memory, motor dexterity) in individuals (ages 12-25) diagnosed with sickle cell disease controlling for age, genotype, and social vulnerability.

Assess the feasibility of an optical imaging tool (Speckle Contrast Optical Spectroscopy - Open-Motion 3.0) to measure cerebral blood flow and blood volume in patients diagnosed with sickle cell disease (ages 12-25).

Assess the concordance between measurement of cerebral blood flow and volume using speckle contrast optical spectroscopy and arterial spin labeling brain MRI.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies

Age range

12 year–25 year

Sex eligibility

All sexes

Study type

Observational

Primary location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location status: Recruiting

Location contact

Andrew Heitzer, PhD

CONTACT

[email protected]

888-226-4343

About this study

Interested participants will be consented in-person or virtually. Consented participants will be mailed the equipment (WatchPAT) to conduct the remote assessment. Additional questionnaires will also be sent via mail or email for the participant to complete prior to collecting data on sleep behaviors. After receiving the questionnaires and equipment, a virtual session will be conducted with the participant to provide education on how to use the equipment and complete the included questionnaires. Participants will wear the WatchPAT overnight for three days and data will be captured remotely. After wearing the devices for three consecutive nights, the participant will attend a research visit within approximately the next two weeks where they will complete performance based neurocognitive assessments, behavioral rating forms, and neuroimaging.

Who can participate

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosed with sickle cell disease of any genotype
  • Participant in the Sickle Cell Clinical Research and Intervention Program
  • Between 12-25 years of age at the time of enrollment
  • English is the primary language
  • Access to an electronic device with WiFi

Exclusion criteria

  • History of an intellectual disability
  • History of a traumatic brain injury or seizure disorder
  • History of a stroke
  • Undergoing potential curative treatment for SCD (stem cell transplant or gene therapy)
  • Currently prescribed an intervention for a sleep disorder
  • Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.

Treatment and study plan

Primary outcomes

  1. Evaluate the relationship between nocturnal oxyhemoglobin saturation (SpO2) and neurocognitive functioning (working memory and verbal comprehension) in individuals (ages 12-25) diagnosed with sickle cell disease.

    Time frame: Baseline remote sleep assessment over 3 days followed by in-clinic assessment.

    A linear regression model will be used to assess the relationship between SpO2 and neurocognitive functioning after adjusting for age, genotype, and social vulnerability.

Secondary outcomes

  1. Assess differences in white matter integrity among individuals (ages 12-25) diagnosed with sickle cell disease with and without sleep disordered breathing.

    Time frame: Baseline remote sleep assessment over 3 days followed by in-clinic assessment.

    This study will measure resting-state BOLD signal in SCD patients after assessing nocturnal oxyhemoglobin saturation. The primary endpoint is the whole brain BOLD response and functional connectivity among brain regions within the default mode, fronto-parietal, executive control, salience, sensorimotor, and other resting state networks. Then we will compare differences in white matter integrity based on the structural connectivity connectome for the working memory network, silent cerebral infarcts, neuroinflammation, and functional connectivity among individuals with and without sleep disordered breathing.

  2. Assess differences in silent cerebral infarcts among individuals (ages 12-25) diagnosed with sickle cell disease with and without sleep disordered breathing.

    Time frame: Baseline remote sleep assessment over 3 days followed by in-clinic assessment.

    This study will measure resting-state BOLD signal in SCD patients after assessing nocturnal oxyhemoglobin saturation. The primary endpoint is the whole brain BOLD response and functional connectivity among brain regions within the default mode, fronto-parietal, executive control, salience, sensorimotor, and other resting state networks. Then we will compare differences in white matter integrity based on the structural connectivity connectome for the working memory network, silent cerebral infarcts, neuroinflammation, and functional connectivity among individuals with and without sleep disordered breathing.

  3. Assess differences in neuroinflammation, among individuals (ages 12-25) diagnosed with sickle cell disease with and without sleep disordered breathing.

    Time frame: Baseline remote sleep assessment over 3 days followed by in-clinic assessment.

    This study will measure resting-state BOLD signal in SCD patients after assessing nocturnal oxyhemoglobin saturation. The primary endpoint is the whole brain BOLD response and functional connectivity among brain regions within the default mode, fronto-parietal, executive control, salience, sensorimotor, and other resting state networks. Then we will compare differences in white matter integrity based on the structural connectivity connectome for the working memory network, silent cerebral infarcts, neuroinflammation, and functional connectivity among individuals with and without sleep disordered breathing.

  4. Assess differences in functional connectivity among individuals (ages 12-25) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age.

    Time frame: Baseline remote sleep assessment over 3 days followed by in-clinic assessment.

    This study will measure resting-state BOLD signal in SCD patients after assessing nocturnal oxyhemoglobin saturation. The primary endpoint is the whole brain BOLD response and functional connectivity among brain regions within the default mode, fronto-parietal, executive control, salience, sensorimotor, and other resting state networks. Then we will compare differences in white matter integrity based on the structural connectivity connectome for the working memory network, silent cerebral infarcts, neuroinflammation, and functional connectivity among individuals with and without sleep disordered breathing.

  5. Assess differences in self- and caregiver-reported mood and pain severity among individuals (ages 12-25) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age.

    Time frame: Baseline remote sleep assessment over 3 days followed by in-clinic assessment.

    The general linear regression model is then used to assess the relationship of functional connectivity and to assess the relationship of self- and caregiver-reported mood and pain severity with sleep disordered breathing with controlling for age in the model. As a secondary analysis, we will also associate continuous obstructive and central AHI with functional connectivity and self- and caregiver-reported mood and pain severity using linear regression model with adjusting for age.

Other outcomes

  1. Explore the association among nocturnal SpO2 and neurocognitive function (attention, processing speed, verbal/visual memory, motor dexterity) in individuals aged 12-25 years with sickle cell disease adjusting for age, genotype, and social vulnerability.

    Time frame: Baseline remote sleep assessment over 3 days followed by in-clinic assessment.

    A linear regression model will be used to assess the relationship between SpO2 and neurocognitive functioning after adjusting for age, genotype, and social vulnerability

  2. Assess the feasibility of an ultraportable ring oximeter (BodimetricsCircul+ Ring) in individuals (ages 12-25) diagnosed with sickle cell disease.

    Time frame: Baseline remote sleep assessment over 3 days followed by in-clinic assessment.

    Feasibility of the Circul+Ring equipment will be defined as more than 50% of participants wearing Circul+Ring for at least one night's sleep. There is no power calculation needed for this feasibility objective. However, 55 participants give 92% power to detect a 20% difference at an assumed 70% of participants who will wear the ring for at least one night's sleep, with a significance level of 0.05 based on a one-sided test. The number of hours worn for the WatchPAT and Circul+Ring will be compared using Wilcoxon rank sum test. Descriptive statistics will be used to summarize the two-question survey.

  3. Assess the concordance between the Circul+Ring with the WatchPAT in individuals (ages 12-25) diagnosed with sickle cell disease.

    Time frame: Baseline remote sleep assessment over 3 days followed by in-clinic assessment.

    To evaluate concordance, the nocturnal SpO2 obtained using the WatchPAT and a Circul+ Ring will be plotted against each other, and Pearson/Spearman correlations will be used to examine their relationship. The Bland-Altman method will be used to investigate the variability between the two measurements using the WatchPAT and Circul+ Ring. Lin's concordance correlation coefficient will be determined to demonstrate the level of agreement.

Study contacts

Contact information is provided by the study sponsor or research team.

Andrew Heitzer, PhD

CONTACT

[email protected]

888-226-4343

Stephanie Guthrie, RN, BSN

CONTACT

[email protected]

888-226-4343

Sponsors and collaborators

Lead sponsor

St. Jude Children's Research Hospital

Other

Registry information

Important dates

Study start
2024
Primary completion
2027
Study completion
2028
First posted
Jun 27, 2024
Registry last updated
Jun 29, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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