Exploring Near Infrared Spectroscopy (NIRS) Technologies for Assessment of Muscle Physiology, Tissue Oxygenation, and Blood Flow in Patients With Sickle Cell Disease (SCD)

NCT05604547 | Completed

• 2 other identifiers: 10000844000844-H
• Study Type: observational
• Target: 49
• Locations: 1 country
• Sites: 1

Brief 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. 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....

Conditions

SCD

Keywords

Hemodynamic; Six-Minute Walk Test

Outcome Measures

Primary Outcomes (1)

• 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

  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.

  120 days

Study Arms (1)

1

Single-arm study

Diagnostic Test: Non-Invasive Infrared Spectroscopy

Interventions

Non-Invasive Infrared Spectroscopy DIAGNOSTIC_TEST

NIRS is a non-Invasive optical technique for characterizing microvascular hemodynamics.

1

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• Sickle Cell Disease
• Individuals with confirmed sickle cell disease (HbSS and S beta 0 genotypes)
• 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
• Individuals with HbAA genotype
• years of age and older
• Willingness and capacity to provide informed consent

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Links

• NIH Clinical Center Detailed Web Page

Study Officials

• Swee Lay Thein, M.D.

  National Heart, Lung, and Blood Institute (NHLBI)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2022
• First Posted: November 3, 2022
• Study Start: November 28, 2022
• Primary Completion: January 24, 2025
• Study Completion: January 24, 2025
• Last Updated: August 1, 2025

Record last verified: 2025-05-13

Locations

US (1)