Clinical trial NCT05283148: Sickle Cell Disease (SCD) Bone Pain Study
Official title: Association Between Low Bone Density, Vertebral Fractures, and Pain in Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: NA
Conditions: Sickle Cell Disease
Sickle Cell Anemia
Low Bone Density
Osteoporosis
Osteopenia
Vertebral Fracture
Vertebral Compression
Osteonecrosis
Ischemic Necrosis
Avascular Necrosis
Keywords: sickle cell disease
sickle cell anemia
low bone density
osteoporosis
osteopenia
low bone mass
Lead sponsor: University of California, Davis
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
name: Doris Duke Charitable Foundation
First posted: 2022-03-16
Enrollment: 53
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Prospective cohort study
Primary purpose: DIAGNOSTIC

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.

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

Primary outcomes:
measure: Bone mineral density Z-scores of lumbar spine, hip, forearm, and whole body
timeFrame: At enrollment
measure: Vertebral fracture analysis
timeFrame: At enrollment
measure: Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) pain scores
timeFrame: At enrollment
Secondary outcomes:
measure: Serum CTX-1
timeFrame: Within 18 months after final enrollment
measure: Serum P1NP
timeFrame: Within 18 months after final enrollment
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: UC Davis Comprehensive Cancer Center
state: California
Study officials:
affiliation: UC Davis School of Medicine
name: Oyebimpe O Adesina, MD, MS
role: PRINCIPAL_INVESTIGATOR
References: