Sickle Cell Disease (SCD) Bone Pain Study

Not Applicable

Active, not recruiting

• Conditions: Sickle Cell Disease; Sickle Cell Anemia; Osteoporosis; Low Bone Density; Vertebral Fracture; Osteopenia; Vertebral Compression; Osteonecrosis; Ischemic Necrosis; Avascular Necrosis
• Interventions: Other: Dual-energy X-ray absorptiometry; Other: Vertebral fracture analysis; Other: Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire

• Registration Number: NCT05283148
• Lead Sponsor: University of California, Davis

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.

Study Timeline

• Study First Submitted: 2022-03-07
• Study First Submitted QC: 2022-03-07
• Study First Posted: 2022-03-16
• Study Start: 2022-11-03
• Primary Completion: 2023-12-22
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-20
• Last Update Posted: 2025-03-25
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 53

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
SCD Bone Pain Study Cohort	Dual-energy X-ray absorptiometry	Prospective cohort of 50 adults with sickle cell disease (SCD) undergoing research DXA scan to assess bone mineral density and thoracolumbar morphometry for vertebral fracture analysis
SCD Bone Pain Study Cohort	Vertebral fracture analysis	Prospective cohort of 50 adults with sickle cell disease (SCD) undergoing research DXA scan to assess bone mineral density and thoracolumbar morphometry for vertebral fracture analysis
SCD Bone Pain Study Cohort	Adult Sickle Cell Quality of Life Measurement System pain impact questionnaire	Prospective cohort of 50 adults with sickle cell disease (SCD) undergoing research DXA scan to assess bone mineral density and thoracolumbar morphometry for vertebral fracture analysis

Primary Outcome Measures

Name	Time	Method
Bone mineral density Z-scores of lumbar spine, hip, forearm, and whole body	At enrollment	Bone mineral density (g/cm2) and standard deviation from reference mean
Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) pain scores	At enrollment	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.
Vertebral fracture analysis	At enrollment	Qualitative measure of thoracolumbar vertebral compression fractures

Secondary Outcome Measures

Name	Time	Method
Serum CTX-1	Within 18 months after final enrollment	Measurement of C-terminal telopeptide of type I collagen (CTX) in serum
Serum P1NP	Within 18 months after final enrollment	Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum

Trial Locations

Locations (1)

UC Davis Comprehensive Cancer Center; 🇺🇸 Sacramento, California, United States