Sickle Cell Disease, Sickle Cell Anemia, Osteonecrosis, Ischemic Necrosis, Avascular Necrosis
Conditions
Keywords
sickle cell disease, sickle cell anemia, osteonecrosis of the femoral head, hip osteonecrosis, ischemic necrosis of the femur, avascular necrosis of the femur
Brief summary
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
Detailed description
The investigators hypothesize that adults with sickle cell disease (SCD) and osteonecrosis of the femoral (hip) and/or humeral (shoulder) heads will tolerate oral alendronate 70 mg administered once a week x 24 weeks (6 months). In addition to collecting safety and tolerability data on alendronate in study participants, the investigators will also measure the preliminary efficacy of alendronate using changes in the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact scores from baseline to 3-months and 6-months after alendronate initiation. Serum and urine specimen will also be collected to assess changes in bone biomarkers at baseline, 3-months, and 6-months. The investigators plan to recruit 24 adults with SCD from the University of California Davis Medical Center to this single-arm, open label, interventional study. The investigators anticipate enrolling 1-2 participants per month between Sep 2024- Dec 2025. The study endpoints are summarized below: * To determine the recruitment and retention rates of adults with SCD and osteonecrosis enrolled in this prospective, single-arm, open label alendronate interventional study * To measure the safety, tolerability, and preliminary efficacy of oral alendronate in adults with SCD-related osteonecrosis over a 6-month treatment duration * To measure changes in bone biomarkers in the serum and urine of study participants not receiving chronic red blood cell transfusions at 3 time points: baseline, 3-months, and 6-months after initiation of alendronate The investigators' goal is to complete primary data analysis by May 2026.
Interventions
Administer oral alendronate 70 mg once a week x 24 weeks to all study participants
Sponsors
University of California, Davis
National Heart, Lung, and Blood Institute (NHLBI)
Doris Duke Charitable Foundation
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Prospective cohort of 30 adults with sickle cell disease and hip osteonecrosis
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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 dual-energy X-ray absorptiometry (DXA) scanner * Negative urine pregnancy test for anyone 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 (for any cause) within 2 weeks of study entry
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Recruitment and retention rates | up to 28 weeks | Proportion of eligible patients who get recruited to the study; number of enrolled patients who complete 6-month study |
| Incidence of Treatment-Emergent Adverse Events as assessed by participant report | up to 28 weeks | Collect all adverse events reported by patients and determine if related to study drug |
| Pain assessed by the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire | up to 28 weeks | The Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire is a patient-reported outcome measure of pain level in the past 7 days. The ASCQ-Me pain scale ranges from 0-100, with a standardized mean of 50 and a standard deviation of 10, where lower scores signify worse disease impact. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Serum CTX-1 | baseline, 3 months, 6 months | Measurement of C-terminal telopeptide of type I collagen (CTX) in serum as a biomarker for osteoclast activity (bone resorption) |
| Serum P1NP | baseline, 3 months, 6 months | Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum as a biomarker for osteoblast activity (bone formation) |
Countries
United States
Contacts
CONTACTLeyla Y Teos, PhD
PRINCIPAL_INVESTIGATOROyebimpe O Adesina, MD, MS
UC Davis School of Medicine
Outcome results
None listed