Trial of Growth Hormone for Osteoporosis Pseudoglioma Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 试验地点
- 2
- 主要终点
- Bone quality by pQCT
研究概览
简要总结
Osteoporosis pseudoglioma (OPPG) syndrome is a rare autosomal recessive condition of childhood osteoporosis and congenital blindness for which new treatments are needed. We have found that body fat is increased in OPPG and muscle mass is reduced. We hypothesize that growth hormone therapy will improve muscle mass and bone strength in OPPG.
详细描述
Osteoporosis pseudoglioma (OPPG) syndrome is a rare autosomal recessive condition of childhood osteoporosis and congenital blindness for which new treatments are needed. We have found that body fat is increased in OPPG and muscle mass is reduced. We hypothesize that growth hormone therapy will improve muscle mass and bone strength in OPPG.
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
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Osteoporosis pseudoglioma syndrome
- •Age 4 years and above
- •not on medication for osteoporosis
排除标准
- •Age under 4 yrs
- •Active malignancy
结局指标
主要结局
Bone quality by pQCT
时间窗: 6 months
By pQCT: periosteal circumference, cortical density, trabecular density, section modulus
次要结局
- Body fat percent(6 months)
研究者
Elizabeth Streeten
Associate Professor of Medicine
University of Maryland, Baltimore