Early Intervention With Zoledronate to Safely Prevent Bone Health Complications in Pediatric Hematopoietic Stem Cell Transplant Survivors
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Safety of early intervention Zoledronate
研究概览
简要总结
The purpose of this pilot study is to investigate the safety and preliminarily assess efficacy of early intervention with zoledronate in high risk pediatric hematopoietic stem cell transplantation (HSCT) patients to prevent the development of bone disease and fractures and reduce potential pain and suffering.
详细描述
Bone disease, including low bone density and fragility fractures (osteoporosis), is common among survivors of pediatric hematopoietic stem cell transplantation (HSCT). Further, patients who develop graft-versus-host disease (GVHD) following HSCT or who have high cumulative doses of glucocorticoids are at even higher risk to develop bone complications. Recent data also suggest that a large number of HSCT candidates arrive to transplantation already with low bone mineral density, adding to the potential risk of developing bone disease following HSCT. Typically, treatment for osteoporosis in children using bisphosphonates, such as zoledronate, is recommended only after the development of fragility fractures.
The investigators propose to study the safety and efficacy of a novel method of early intervention with zoledronate in high risk pediatric HSCT patients to prevent the development of bone disease and fractures in order to reduce potential pain and suffering.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients ≥5 and ≤18 years old who are preparing for HSCT with a height-for-age corrected DXA Z-score of <-2.0 and admitted to a CCHMC inpatient unit.
- •Patients ≥5 and ≤18 years old recovering from HSCT and who have developed de novo acute or chronic GVHD and are admitted to a CCHMC inpatient unit.
排除标准
- •Age <5 years and >18 years
- •Patients with Fanconi anemia or other radiation-sensitive syndromes with increased malignancy risk
- •history of prior bisphosphonate use
- •low 25-OH vitamin D levels (<20 ng/mL)
- •active febrile illness
- •uncontrolled infection
- •Elevated creatinine at the time of enrollment, history or renal failure, or documented low glomerular filtration rate (GFR≤90)
- •Active bone disease including history of abnormal PTH level for any reason, active bone fracture/healing, or primary disorder of bone development or metabolism.
- •Women who are pregnant or breast feeding.
研究组 & 干预措施
Zoledronate
Zoledronate dose will be given intravenously over 1 hour using standard infusion equipment. Patients will receive a dose of 0.025 mg/kg. Zoledronate will be infused in the inpatient setting to facilitate proper observation and data collection.
干预措施: Zoledronate (Drug)
结局指标
主要结局
Safety of early intervention Zoledronate
时间窗: At the time of infusion through up to 60 days post last dose of Zoledronate
Calcium levels following infusion of zoledronate
Feasibility of early intervention Zoledronate
时间窗: At patient discharge, typically 1 week post infusion
Number of days required for inpatient stay beyond what is necessary for the patient's admission
次要结局
- Bone health efficiency(Baseline, +6 months, and +12 months)
- Bone substrate turnover(Baseline and +30 days)