Effects of Theophylline on cAMP Signaling in Children With Pseudohypoparathyroidism Type 1a
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Change in Urine cAMP
研究概览
简要总结
This study will test an investigational drug, theophylline, in children with pseudohypoparathyroidism type 1a (PHP1a). This study involves a 3 day visit to the Vanderbilt Clinical Research Center.
详细描述
Study measures will be done at baseline and during a 24h infusion of IV theophylline. Theophylline levels will be drawn to ensure therapeutic dosing and to monitor for toxicity. Measures include laboratory values, response to PTH infusion and resting energy expenditure.
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
- 10 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 10 to 21 years old
- •English proficiency
- •Clinical and genetic diagnosis of PHP1a
排除标准
- •Use of a PDE inhibitor in the past 30 days
- •History of a seizure disorder unrelated to hypocalcemia
- •History of a cardiac arrhythmia (not including bradycardia)
- •History of hepatic insufficiency
- •AST or ALT >2x upper limit of normal
- •Total bilirubin >1.5 x upper limit of normal (unless patient has a diagnosis of Gilbert's syndrome and no other causes leading to hyperbilirubinemia are identified)
- •Congestive heart failure
- •Cigarette use in the past 30 days
- •Alcohol use within the past 24 hours
- •Current pregnancy
- •Untreated hypothyroidism (defined as free T4 level < 0.6 ng/dL or TSH >10 mcU/mL)
- •Active peptic ulcer disease
- •Fever >101 degrees in the past 24 hours
- •Current use of medications known to effect theophylline levels (listed below)
- •Severe sleep apnea requiring BiPAP
- •Drugs with clinically significant drug interactions with theophylline:
- •Allopurinol
- •Cimetidine
- •Ciprofloxacin
- •Clarithromycin
- •Ephedrine
- •Erythromycin
- •Flurazepam
- •Fluvoxamine
- •Lorazepam
- •Methotrexate
- •Midazolam
- •Pentoxifylline
- •Propranolol
- •Sulfinpyrazone
- •Thiabendazole
- •Ticlopidine
- •Troleandomycin
- •Verapamil
研究组 & 干预措施
Theophylline
Patients will receive a 24 hour continuous infusion of intravenous theophylline.
干预措施: Theophylline (Drug)
结局指标
主要结局
Change in Urine cAMP
时间窗: 1 day
Change in urine cAMP (after parathyroid hormone stimulation) before and during treatment with theophylline
次要结局
- Change in Apnea Hypopnea Index (AHI)(1 day)
- Change in Resting Energy Expenditure (REE)(1 day)
研究者
Ashley Shoemaker
Assistant Professor
Vanderbilt University Medical Center