Sickle Cell Disease (SCD) Bone Pain Study

Osteoporosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05283148
• Other study ID #: 1782537
• Secondary ID: K23HL14831020200
• Status: Active, not recruiting
• Phase: N/A
• Start date: November 3, 2022
• Est. completion date: March 31, 2024

Study information

• Verified date: September 2023
• Source: University of California, Davis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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- Oct 2023, and anticipate enrolling 4-6 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 53
• Est. completion date: March 31, 2024
• Est. primary completion date: December 22, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Avascular Necrosis
• Bone Diseases, Metabolic
• Low Bone Density
• Necrosis
• Osteonecrosis
• Osteopenia
• Osteoporosis
• Sickle Cell Anemia
• Sickle Cell Disease
• Spinal Fractures
• Vertebral Compression
• Vertebral Fracture

Intervention

Other:
• Dual-energy X-ray absorptiometry
Measure bone mineral density at the lumbar spine, left total hip, left forearm, and whole body
• Vertebral fracture analysis
Obtain thoracolumbar morphometry in DXA scan, then determine presence and severity of vertebral compression fractures by VFA
• Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire
Calculate patient-reported total pain scores to determine the pain phenotype of each study participant

Locations

Country	Name	City	State
United States	UC Davis Comprehensive Cancer Center	Sacramento	California

Sponsors (3)

Lead Sponsor	Collaborator
University of California, Davis	Doris Duke Charitable Foundation, National Heart, Lung, and Blood Institute (NHLBI)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Bone mineral density Z-scores of lumbar spine, hip, forearm, and whole body	Bone mineral density (g/cm2) and standard deviation from reference mean	At enrollment
Primary	Vertebral fracture analysis	Qualitative measure of thoracolumbar vertebral compression fractures	At enrollment
Primary	Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) pain scores	Patient-reported outcome measure of pain level in the past 7 days. The ASCQ-Me pain scale ranges from 0-100, with a standardized sickle cell disease population mean of 50 (standard deviation=10), where lower scores signify worse disease impact.	At enrollment
Secondary	Serum CTX-1	Measurement of C-terminal telopeptide of type I collagen (CTX) in serum	Within 18 months after final enrollment
Secondary	Serum P1NP	Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum	Within 18 months after final enrollment