A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Administer oral alendronate 70 mg once a week x 24 weeks to all study participants
UC Davis Comprehensive Cancer Center
Sacramento, California, United States
RECRUITINGRecruitment and retention rates
Proportion of eligible patients who get recruited to the study; number of enrolled patients who complete 6-month study
Time frame: up to 28 weeks
Incidence of Treatment-Emergent Adverse Events as assessed by participant report
Collect all adverse events reported by patients and determine if related to study drug
Time frame: up to 28 weeks
Pain assessed by the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me) Pain Impact questionnaire
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.
Time frame: up to 28 weeks
Serum CTX-1
Measurement of C-terminal telopeptide of type I collagen (CTX) in serum as a biomarker for osteoclast activity (bone resorption)
Time frame: baseline, 3 months, 6 months
Serum P1NP
Measurement of Procollagen type I N-terminal propeptide (P1NP) in serum as a biomarker for osteoblast activity (bone formation)
Time frame: baseline, 3 months, 6 months
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