Effect of Theophylline in Pseudohypoparathyroidism
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Effect of Theophylline on Bone Age in PHP
研究概览
简要总结
The study evaluates the effect of theophylline in 100 subjects with Pseudohypoparathyroidism.
详细描述
It is an open label intervention study for the off-label use of theophylline in psuedohypoparathyroidism (PHP).Theophylline is a is non-specific phosphodiesterase inhibitor which increases the levels of secondary messenger,cAMP. PHP patients have end organ resistance to hormones involving cAMP signaling. Thus, theophylline has a potential to abrogate hormone resistance. Also, it may have a role in bone maturation, neurocognition and metabolism.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects with known diagnosis of PHP1A and PHP1B
- •Age >5 years
排除标准
- •• Significant kidney disease, i.e. any CKD stage
- •Hypercalciuria
- •Kidney stones
- •Severe Heart disease
- •Uncontrolled seizure disorder
- •Peptic ulcer
- •Liver dysfunction
- •Bleeding disorder
- •Allergy to theophylline preparation
- •Muscle disorder
- •Infection
- •Any neurologic disease
- •Chronic drugs interacting with theophylline
研究组 & 干预措施
Single arm open labeled intervention study
Subjects with PHP will be given theophylline to decrease the end organ resistance by increasing levels of cAMP, a second messenger. Theophylline will be dosed twice a day for a period of 52 weeks.
干预措施: Theophylline (Drug)
结局指标
主要结局
Effect of Theophylline on Bone Age in PHP
时间窗: 52 weeks
Change in Bone age ( in years and months) before and after theophylline administration for 52 weeks
次要结局
未报告次要终点
研究者
Amita Sharma
Medical Director, Division of Pediatric Nephrology
Massachusetts General Hospital