A Feasibility Study of Alendronate as Treatment for Osteonecrosis in Adults With Sickle Cell Disease
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Incidence of Treatment-Emergent Adverse Events as assessed by participant report
研究概览
简要总结
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
Alendronate group
Single-arm prospective cohort of 30 adult with SCD
干预措施: Alendronate Sodium (Drug)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events as assessed by participant report
时间窗: 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
时间窗: 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
时间窗: up to 28 weeks
Proportion of eligible patients who get recruited to the study; number of enrolled patients who complete 6-month study
次要结局
- Serum CTX-1(baseline, 3 months, 6 months)
- Serum P1NP(baseline, 3 months, 6 months)
- Bone turnover markers(Baseline, then every 2 months until end of study)
- Inflammatory biomarkers(Baseline, then every 2 months until the end of the study)
- Vascular adhesion biomarkers(Baseline, then every 2 months until the end of the study)