SCDNCT05604547
Exploring Near Infrared Spectroscopy (NIRS) Technologies for Assessment of Muscle Physiology, Tissue Oxygenation, and Blood Flow in Patients With Sickle Cell Disease (SCD)
An observational study of SCD, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Sickle cell disease (SCD) is an inherited disorder of the blood. SCD can injure the smallest blood vessels, which can cause pain and damage organs all over the body. Some treatments are available, but researchers need better ways to monitor the effects of these treatments. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- SCD
- What is being tested?
- Non-Invasive Infrared Spectroscopy
- How many people
- 49 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Nov 2022to 24 Jan 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: We aim to enroll 20 individuals in each of the following participant groups: SCD (20 HbSS/S-beta0) and 20 healthy controls (genotype HbAA) To obtain this level of enrollment we request a screening ceiling of 60 individuals (30 people per each group) for an enrolment target of 20 SCD and 20 healthy controls who complete participation. Twenty individuals per group should help identify specific hypotheses for testing and estimates of variability and effect size. Correlations between NIRS measurements and clinical/biomarker measurements will need to be relatively strong to meet statistical significance criteria at a 5 percent alpha level for a two-sided test. Assuming data have a bivariate normal distribution, a true correlation of 0.5 and 0.6 are associated with power of 64 percent and 84 percent, respectively. If a Spearman correlation test is used instead of a Pearson correlation (if distribution appear non-normal) then power falls to 58 percent and 76 percent, respectively.
Who may be able to join
- Sickle Cell Disease
- Individuals with confirmed sickle cell disease (HbSS and S beta 0 genotypes)
- 18 years of age and older
- Willingness and capacity to provide informed consent
- Individuals must be on stable does of Hydroxyurea for 90 days prior to baseline visit
- Ethnically matched controls
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- Individuals with HbAA genotype
- 18 years of age and older
- Willingness and capacity to provide informed consent
Who cannot join
- Sickle Cell Disease
- Pain crisis requiring parenteral treatment within 4 weeks of screening/enrollment
- Blood transfusion within 60 days or exchange transfusion within 90 days of screening/enrollment
- Use of Oxbryta (voxelotor), Adakveo (crizanlizumab), and/or Endari (L-glutamine) within the 12 weeks prior to signing consent
- Women who are currently pregnant
- Presence of an arterial-venous shunt, recent axillary node dissection, or any deformity or surgical history that interferes with proper access for brachial cuff occlusion procedure of the extremities
Show all 12Show fewer
- Mobility difficulties causing the inability to complete 6-minute walk test
- Ethnically matched controls
- Women who are currently pregnant
- Presence of an arterial-venous shunt, recent axillary node dissection, or any deformity or surgical history that interferes with proper access for brachial cuff occlusion procedure of the extremities
- Mobility difficulties causing the inability to complete 6-minute walk test
- Blood transfusion within 60 days or exchange transfusion within 90 days of screening/enrollment
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Sickle cell disease (SCD) is an inherited disorder of the blood. SCD can injure the smallest blood vessels, which can cause pain and damage organs all over the body. Some treatments are available, but researchers need better ways to monitor the effects of these treatments. An imaging technique called near infrared spectroscopy (NIRS) may be helpful.
Objective:
Read more
To test NIRS as a tool for measuring oxygen levels, blood flow, and the makeup of skin and muscle in patients with SCD.
Eligibility:
People aged 18 years and older with SCD. Healthy volunteers are also needed as a comparison for the changes in SCD patients.
Design:
Participants will be screened. They will have a physical exam, and 1 teaspoon of blood will be drawn.
Participants will have NIRS testing on their second visit. Probes will be placed on their skin. A blood pressure cuff will be placed on their arm. The cuff will be filled with air for up to 5 minutes and then released. Participants may be asked to breathe at a certain rate or hold their breath during these measurements.
At this visit, participants will also have an ultrasound exam to get images of their heart. They will be monitored while they walk for 6 minutes. They will have 1 tablespoon of blood drawn. Their height, weight, and vital signs will be measured.
Participants may be asked to return for up to 4 additional visits for NIRS testing within 120 days, but this is optional. The visits must be at least 3 days apart. Each visit will last up to an hour....
Read the detailed description
Study Description:
This is a non-interventional protocol to explore the role of near infrared spectroscopy (NIRS) technologies as a monitoring tool for assessment of peripheral tissue dynamics in patients with sickle cell disease (SCD). The study will be performed in the outpatient setting. Ethnically-matched healthy subjects will be recruited to participate in baseline NIRS assessments and serve as controls. It is hypothesized that quantitative NIRS will be sensitive to differences in hemodynamic responses between SCD patients and healthy controls in the setting of endothelial dysfunction. In parallel, routine methods of assessing cardiovascular health, including the six-minute walk test, transthoracic echocardiography, and cardiac biomarkers such as pro BNP will be acquired. NIRS holds tremendous potential as a novel point of care test in patients with SCD and may provide further insight on the natural history of the disease and allow for individualized clinical management.
A subset of up to 10 participants in each cohort (SCD patients as well as healthy AA subjects) may be invited to return to the Clinical Center for clinical and NIRS reassessment periodically for a maximum of 4 additional visits, at least 3 days apart, within a 120- day period from their initial (baseline) visit to test repeatability of the NIRS measurements.
Objectives:
Primary Objective:
To quantify baseline hemodynamics in SCD subjects at steady state utilizing NIRS and compare against established clinical/biomarker outcomes, including the six-minute walk test, transthoracic echocardiogram and pro BNP levels.
Secondary Objective:
To compare NIRS measurements in SCD subjects at steady state with NIRS measurements in healthy controls.
Tertiary Objective:
To quantify baseline hemodynamics in ethnically-matched healthy control subjects (HbAA) utilizing NIRS and compare against established clinical/biomarker outcomes, including the six-minute walk test, transthoracic echocardiogram and pro BNP levels.
Endpoints:
Primary Endpoints:
NIRS measurements that characterize tissue composition, tissue metabolism, and blood flow will be collected in SCD subjects in steady state. Primary endpoints include:
- Oxy-/deoxy- hemoglobin concentration for assessing tissue composition
- Tissue oxygen saturation for assessing tissue metabolism
- Blood flow index: Post-occlusion hyperemic response for describing endothelial function.
We will examine correlations between these endpoints and biomarker and clinical endpoints among SCD participants to assess the extent to which NIRS measurements may capture clinical features of SCD.
Secondary Endpoints:
The same endpoints described above will be collected from healthy controls and used to assess differences in tissue composition, tissue metabolism, blood flow between SCD subjects and controls.
Tertiary Endpoints:
To evaluate the tertiary objective of characterizing NIRS responses in healthy controls we will collect:
- Oxy-/deoxy- hemoglobin concentration for assessing tissue composition
- Tissue oxygen saturation for assessing tissue metabolism
- Blood flow index: Post-occlusion hyperemic response for describing endothelial function.
- Conditions
- SCD
- Being tested
- Non-Invasive Infrared Spectroscopy
- Keywords
- Hemodynamic, Six-Minute Walk Test
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non-Invasive Infrared SpectroscopyDiagnostic test
NIRS is a non-Invasive optical technique for characterizing microvascular hemodynamics.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Single-arm study Receives: Diagnostic Test: Non-Invasive Infrared Spectroscopy |
What is being measured
The primary outcome is the main question the study is designed to answer.
To quantify baseline hemodynamic function in SCD subjects at steady state utilizing NIRS in parallel with clinical assessments of cardiovascular function, including the 6-minute walk test (6MWT), pro BNP levels and transthoracic echocardiogram.
Baseline NIRS measurements will permit real time assessment of peripheral tissue hemodynamics, including oxygenation and blood flow, in relation to established markers of cardiovascular health in subjects with SCD, including the 6MWT, TTE, and pro BNP levels
Measured: 120 days
Secondary outcomes (1)
To assess baseline tissue hemodynamic function utilizing NIRS in ethnically-matched healthy control subjects without (AA) and with sickle cell trait (AS) as compared to baseline NIRS assessments in patients with SCD.
This will permit comparison of peripheral tissue hemodynamics, including blood flow and oxygenation, and cardiovascular clinical outcomes (6MWT, TTE, pro BNP) in subjects with SCD and ethnically-matched healthy volunteer subjects. NIRS results may validate abnormal tissue hemodynamic responses observed in subjects with SCD as compared to controls
Measured: 120 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Nov 2022
- Study start28 Nov 2022
- Primary completion24 Jan 2025
- Study completion24 Jan 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SCDStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Swee Lay Thein, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05604547
- Sponsor's protocol ID
- 10000844
- Other IDs
- 000844-H
- Status last verified
- 13 May 2025
- First submitted
- 2 Nov 2022
- Last updated
- 1 Aug 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Undecided: It is not yet known if there will be a plan to make IPD available
Study at a glance
From the registry record, last updated by the sponsor on 1 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploring Near Infrared Spectroscopy (NIRS) Technologies for Assessment of Muscle Physiology, Tissue Oxygenation, and Blood Flow in Patients With Sickle Cell Disease (SCD)
- ClinicalTrials.gov ID
- NCT05604547
- Acronym / sponsor ID
- 10000844
- Conditions
- SCD
- Keywords
- Hemodynamic, Six-Minute Walk Test
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 28 Nov 2022 (actual)
- Primary completion
- 24 Jan 2025 (actual)
- Study completion
- 24 Jan 2025 (actual)
- First posted
- 3 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Sickle cell disease (SCD) is an inherited disorder of the blood. SCD can injure the smallest blood vessels, which can cause pain and damage organs all over the body. Some treatments are available, but researchers need better ways to monitor the effects of these treatments. An imaging technique called near infrared spectroscopy (NIRS) may be helpful.
Objective:
To test NIRS as a tool for measuring oxygen levels, blood flow, and the makeup of skin and muscle in patients with SCD.
Eligibility:
People aged 18 years and older with SCD. Healthy volunteers are also needed as a comparison for the changes in SCD patients.
Design:
Participants will be screened. They will have a physical exam, and 1 teaspoon of blood will be drawn.
Participants will have NIRS testing on their second visit. Probes will be placed on their skin. A blood pressure cuff will be placed on their arm. The cuff will be filled with air for up to 5 minutes and then released. Participants may be asked to breathe at a certain rate or hold their breath during these measurements.
At this visit, participants will also have an ultrasound exam to get images of their heart. They will be monitored while they walk for 6 minutes. They will have 1 tablespoon of blood drawn. Their height, weight, and vital signs will be measured.
Participants may be asked to return for up to 4 additional visits for NIRS testing within 120 days, but this is optional. The visits must be at least 3 days apart. Each visit will last up to an hour....
Detailed description
Study Description:
This is a non-interventional protocol to explore the role of near infrared spectroscopy (NIRS) technologies as a monitoring tool for assessment of peripheral tissue dynamics in patients with sickle cell disease (SCD). The study will be performed in the outpatient setting. Ethnically-matched healthy subjects will be recruited to participate in baseline NIRS assessments and serve as controls. It is hypothesized that quantitative NIRS will be sensitive to differences in hemodynamic responses between SCD patients and healthy controls in the setting of endothelial dysfunction. In parallel, routine methods of assessing cardiovascular health, including the six-minute walk test, transthoracic echocardiography, and cardiac biomarkers such as pro BNP will be acquired. NIRS holds tremendous potential as a novel point of care test in patients with SCD and may provide further insight on the natural history of the disease and allow for individualized clinical management.
A subset of up to 10 participants in each cohort (SCD patients as well as healthy AA subjects) may be invited to return to the Clinical Center for clinical and NIRS reassessment periodically for a maximum of 4 additional visits, at least 3 days apart, within a 120- day period from their initial (baseline) visit to test repeatability of the NIRS measurements.
Objectives:
Primary Objective:
To quantify baseline hemodynamics in SCD subjects at steady state utilizing NIRS and compare against established clinical/biomarker outcomes, including the six-minute walk test, transthoracic echocardiogram and pro BNP levels.
Secondary Objective:
To compare NIRS measurements in SCD subjects at steady state with NIRS measurements in healthy controls.
Tertiary Objective:
To quantify baseline hemodynamics in ethnically-matched healthy control subjects (HbAA) utilizing NIRS and compare against established clinical/biomarker outcomes, including the six-minute walk test, transthoracic echocardiogram and pro BNP levels.
Endpoints:
Primary Endpoints:
NIRS measurements that characterize tissue composition, tissue metabolism, and blood flow will be collected in SCD subjects in steady state. Primary endpoints include:
- Oxy-/deoxy- hemoglobin concentration for assessing tissue composition
- Tissue oxygen saturation for assessing tissue metabolism
- Blood flow index: Post-occlusion hyperemic response for describing endothelial function.
We will examine correlations between these endpoints and biomarker and clinical endpoints among SCD participants to assess the extent to which NIRS measurements may capture clinical features of SCD.
Secondary Endpoints:
The same endpoints described above will be collected from healthy controls and used to assess differences in tissue composition, tissue metabolism, blood flow between SCD subjects and controls.
Tertiary Endpoints:
To evaluate the tertiary objective of characterizing NIRS responses in healthy controls we will collect:
- Oxy-/deoxy- hemoglobin concentration for assessing tissue composition
- Tissue oxygen saturation for assessing tissue metabolism
- Blood flow index: Post-occlusion hyperemic response for describing endothelial function.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | Diagnostic Test: Non-Invasive Infrared SpectroscopySingle-arm study |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- We aim to enroll 20 individuals in each of the following participant groups: SCD (20 HbSS/S-beta0) and 20 healthy controls (genotype HbAA) To obtain this level of enrollment we request a screening ceiling of 60 individuals (30 people per each group) for an enrolment target of 20 SCD and 20 healthy controls who complete participation. Twenty individuals per group should help identify specific hypotheses for testing and estimates of variability and effect size. Correlations between NIRS measurements and clinical/biomarker measurements will need to be relatively strong to meet statistical significance criteria at a 5 percent alpha level for a two-sided test. Assuming data have a bivariate normal distribution, a true correlation of 0.5 and 0.6 are associated with power of 64 percent and 84 percent, respectively. If a Spearman correlation test is used instead of a Pearson correlation (if distribution appear non-normal) then power falls to 58 percent and 76 percent, respectively.
Key inclusion criteria (9 listed)
- Sickle Cell Disease
- Individuals with confirmed sickle cell disease (HbSS and S beta 0 genotypes)
- 18 years of age and older
- Willingness and capacity to provide informed consent
- Individuals must be on stable does of Hydroxyurea for 90 days prior to baseline visit
Key exclusion criteria (12 listed)
- Sickle Cell Disease
- Pain crisis requiring parenteral treatment within 4 weeks of screening/enrollment
- Blood transfusion within 60 days or exchange transfusion within 90 days of screening/enrollment
- Use of Oxbryta (voxelotor), Adakveo (crizanlizumab), and/or Endari (L-glutamine) within the 12 weeks prior to signing consent
- Women who are currently pregnant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Non-Invasive Infrared SpectroscopyNIRS is a non-Invasive optical technique for characterizing microvascular hemodynamics. | Diagnostic test | 1 |
Primary outcome
To quantify baseline hemodynamic function in SCD subjects at steady state utilizing NIRS in parallel with clinical assessments of cardiovascular function, including the 6-minute walk test (6MWT), pro BNP levels and transthoracic echocardiogram.
Time frame: 120 days
Baseline NIRS measurements will permit real time assessment of peripheral tissue hemodynamics, including oxygenation and blood flow, in relation to established markers of cardiovascular health in subjects with SCD, including the 6MWT, TTE, and pro BNP levels
1 secondary outcome
To assess baseline tissue hemodynamic function utilizing NIRS in ethnically-matched healthy control subjects without (AA) and with sickle cell trait (AS) as compared to baseline NIRS assessments in patients with SCD.
Time frame: 120 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Aug 2025.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided. Undecided: It is not yet known if there will be a plan to make IPD available
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Swee Lay Thein, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of SCD, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To quantify baseline hemodynamic function in SCD subjects at steady state utilizing NIRS in parallel with clinical assessments of cardiovascular function, including the 6-minute walk test (6MWT), pro BNP levels and transthoracic echocardiogram. (time frame: 120 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Aug 2025.