Alendronate for Osteonecrosis in Adults With Sickle Cell Disease

Phase 2

Not yet recruiting

• Conditions: Sickle Cell Disease; Sickle Cell Anemia; Osteonecrosis; Avascular Necrosis; Ischemic Necrosis
• Interventions: Drug: Alendronate Sodium

• Registration Number: NCT06016634
• Lead Sponsor: University of California, Davis

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.

Study Timeline

• Study First Submitted: 2023-08-07
• Study First Submitted QC: 2023-08-22
• Study First Posted: 2023-08-30
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-21
• Last Update Posted: 2025-07-25
• Study Start: 2025-09-01
• Primary Completion: 2026-12-31
• Study Completion: 2027-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 30

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Alendronate group	Alendronate Sodium	Single-arm prospective cohort of 30 adult with SCD

Primary Outcome Measures

Name	Time	Method
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.
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

Secondary Outcome Measures

Name	Time	Method
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)

Trial Locations

Locations (1)

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