Bone and Joint Health in an Adult Hemophilia Population
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- Longitudinal Endpoint: Change in bone mineral density by DXA over 2 years
研究概览
简要总结
The purpose of this study is to evaluate bone mineral density in adult subjects with hemophilia versus a comparator population without hemophilia (non-hemophilia age- and gender-matched database) by using the following diagnostic means: dual-energy X-ray absorptiometry (DXA) scanning, clinical scales, quality of life (QOL) scales and biomarkers. In addition to this osteoporosis study, hemophilic arthropathy of the knee with respect to loss of knee cartilage will also be explored by using magnetic resonance imaging (MRI substudy). No investigational product will be dispensed.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 25 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male and at least 25 years old at the time of screening
- •Moderately severe or severe hemophilia A or B (factor VIII or factor IX <= 2% by chart documentation)
- •Ambulatory (ie, not wheel chair dependent)
- •Performance status - ECOG of 0 or 1 (= out of bed at least 75% of time)
- •Willing and able to comply with the requirements of the protocol and is able to give informed consent
- •Additional inclusion criteria for MRI Substudy:
- •Baseline knee joint space width is >= 3mm at the medial or lateral aspect of either knee as measured by weightbearing fixed flexion PA X-ray
- •Able to undergo 1.5 or 3T MRI
排除标准
- •Inability to position properly for DXA
- •Presence of orthopedic hardware or other artifact in the lumbar spine (L1-L4) or either proximal femur
- •Prior documentation of being HIV positive
- •Radiosynovectomy or surgical synovectomy within the last 6 months
- •Current or past treatment with bone active drugs
- •Long-term corticosteroid use [defined as 7.5-mg prednisone daily (or equivalent) for >= 3 months]
- •If subject is family member or employee of the investigator
- •Additional exclusion criteria for MRI Substudy:
- •Any contraindication or relative contraindication to MRI
- •Less than 3mm knee joint space width at the medial and lateral aspect of both knees on baseline X-ray evaluation
- •Knee bleed within 30 days of informed consent
- •Prior total knee arthroplasty (TKA)
结局指标
主要结局
Longitudinal Endpoint: Change in bone mineral density by DXA over 2 years
时间窗: 2 years
Longitudinal Endpoint: Rate of loss of knee cartilage parameters over 2 years by MRI
时间窗: 2 years
Baseline Endpoint: Z-Score by dual-energy X-ray absorptiometry (DXA) at baseline
时间窗: Baseline
次要结局
未报告次要终点