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Clinical Trials/NCT05283148
NCT05283148
Active, not recruiting
Not Applicable

Association Between Low Bone Density, Vertebral Fractures, and Pain in Sickle Cell Disease

University of California, Davis1 site in 1 country53 target enrollmentNovember 3, 2022

Overview

Phase
Not Applicable
Intervention
Not specified
Conditions
Sickle Cell Disease
Sponsor
University of California, Davis
Enrollment
53
Locations
1
Primary Endpoint
Lumbar Spine Bone Mineral Density
Status
Active, not recruiting
Last Updated
8 months ago

Overview

Brief Summary

A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease

Detailed Description

The investigators hypothesize that adults with sickle cell disease (SCD) and low bone density and/or vertebral compression fractures on a dual X-ray absorptiometry (DXA) scan (adjusted for age, sex, SCD genotype, relevant labs, presence of osteonecrosis, and SCD-modifying therapies) will report more severe pain than those with normal bone density or no vertebral fractures. The Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) is a validated patient-reported outcome measure of physical, mental, and social health in adults with SCD. This cross-sectional observational study involves obtaining a baseline DXA scan, vertebral fracture analysis (VFA) and pain assessment using ASCQ-Me pain impact scores. The investigators plan to recruit 50 adults with SCD followed at University of California Davis Medical Center between Nov 2022- Dec 2023 and anticipate enrolling up to 4 adults with SCD per month. The study endpoints are listed below: * To determine the association between bone density Z-scores and ASCQ-Me pain impact scores in a prospective cohort of adults with SCD * To study the association between Spine Deformity Index scores (SDI, a proxy for vertebral fracture analysis) and ASCQ-Me pain impact scores in a prospective cohort of adults with SCD * To assess the correlation between baseline hematological and biochemical laboratory parameters (including bone biomarkers), bone density, and/or vertebral fractures in a prospective cohort of adults with SCD The investigators' goal is to complete primary data analysis by Mar 2024. As an exploratory endpoint, 1cc of serum and 5cc of urine will be collected from each study participant once (at baseline), after an overnight fast, for bone biomarker analyses.

Registry
clinicaltrials.gov
Start Date
November 3, 2022
End Date
July 31, 2026
Last Updated
8 months ago
Study Type
Interventional
Study Design
Single Group
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • Age 18-80 years with SCD (any genotype, confirmed by hemoglobin electrophoresis or high performance liquid chromatography)
  • Ability to provide written informed consent
  • Ability to lay on a DXA scanner
  • Negative urine pregnancy test for women of childbearing potential at study entry

Exclusion Criteria

  • Pregnant women
  • Adults unable to consent
  • Individuals who are not yet adults (infants, children, teenagers)
  • Prisoners
  • Hospitalizations (any cause) within 2 weeks of study entry

Outcomes

Primary Outcomes

Lumbar Spine Bone Mineral Density

Time Frame: Baseline

Areal bone mineral density of the lumbar spine measured by dual-energy X-ray absorptiometry (DXA) scan and reported in grams per square centimeter.

Femoral Neck Bone Mineral Density (BMD)

Time Frame: Baseline

Areal bone mineral density of the femoral neck measured by dual-energy X-ray absorptiometry (DXA) scan and reported in grams per square centimeter.

Femoral Neck Bone Mineral Density Z-scores

Time Frame: Baseline

Number of standard deviations between measured lumbar spine bone mineral density (g/cm2) for each participant and mean lumbar spine bone mineral density (g/cm2) of the reference population. A Z-score of 0 represents the mean of the reference population. A negative Z-score means the measured bone mineral density values are lower (worse) the reference mean, while positive Z-scores mean they are above/higher. Bone mineral density Z-scores ≤ -2 indicates low bone density.

Lumbar Spine Bone Mineral Density-Z-scores

Time Frame: Baseline

Number of standard deviations between measured lumbar spine bone mineral density (g/cm2) for each participant and mean lumbar spine bone mineral density (g/cm2) of the reference population. A Z-score of 0 represents the mean of the reference population. A negative Z-score means the measured bone mineral density values are lower (worse) the reference mean, while positive Z-scores mean they are above/higher. Bone mineral density Z-scores ≤ -2 indicates low bone density.

Total Hip Bone Mineral Density (BMD)

Time Frame: Baseline

Areal bone mineral density of the total hip measured by dual-energy X-ray absorptiometry (DXA) scan and reported in grams per square centimeter.

Total Hip Bone Mineral Density-Z-scores

Time Frame: At enrollment

Number of standard deviations between measured total hip bone mineral density (g/cm2) for each participant and mean total hip bone mineral density (g/cm2) of the reference population. A Z-score of 0 represents the mean of the reference population. A negative Z-score means the measured bone mineral density values are lower (worse) the reference mean, while positive Z-scores mean they are above/higher. Bone mineral density Z-scores ≤ -2 indicates low bone density.

Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact T-scores

Time Frame: Baseline

Patient-reported outcome measure of pain impact in the preceding 7 days before bone density measurements. The Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) pain impact T-scores range from about 30-100. The ASCQ-Me pain impact T-score has standardized mean T-score of 50 and standard deviation of 10, which were derived from a reference population of ambulatory adult with sickle cell disease across the United States. ASCQ-Me pain impact T-scores less than 50 are lower/worse than the reference mean (more severe pain impact), while pain impact T-scores greater than 50 are above/better than the reference mean (less severe pain impact)

Secondary Outcomes

  • Spinal Deformity Index(Baseline)

Study Sites (1)

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