Kuvan® (Sapropterin Dihydrochloride) for Improving Gastric Accommodation in Women With Diabetic Gastroparesis (KIGA-DG)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Gastric Accommodation
研究概览
简要总结
Kuvan® (sapropterin dihydrochloride) for Improving Gastric Accommodation in Women with Diabetic Gastroparesis (KIGA-DG)
详细描述
Patients are invited to participate in a research study of Kuvan® (sapropterin dihydrochloride). We hope to learn whether treatment with Kuvan® is safe and effective in improving the ability of the stomach to relax after eating and improving the symptoms of diabetic gastroparesis. Women are selected as a possible participant in this study because they have diabetes and moderate to severe gastroparesis (meaning stomach empties slowly).
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
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Inclusion Criteria
- •Patients with moderate to severe symptoms of diabetic gastroparesis will be studied (GCSI >21). Patients should not have post-surgical gastroparesis or dyspeptic symptoms with normal gastric emptying. In order to qualify for inclusion in the trial, patients must satisfy the following inclusion criteria:
- •Diagnosis of diabetes mellitus > 5 years requiring medical therapy
- •Female gender
- •Ages 18-65 years
- •Documentation of delayed gastric emptying on gastric emptying scintigraphy (within 2 years of enrollment)
- •Symptoms of gastroparesis for at least 6 months with Gastroparesis Cardinal Symptom Index (GCSI) score > 21 indicating moderate to severe symptoms
- •Recent negative upper endoscopy or upper GI series within 2 years of enrollment (no evidence of mechanical obstruction or peptic ulcer disease)
排除标准
- •Exclusion Criteria
- •Patients who satisfy any of the following exclusion criteria will be ineligible for enrollment in the study:
- •Diabetes diagnosed < 5 years prior to the study
- •Male gender
- •Normal gastric emptying
- •Gastroparesis from post-surgical etiologies
- •Another active disorder, which could explain symptoms in the opinion of the investigator
- •History of significant cardiac arrhythmias and/or prolonged QTc
- •Daily use of narcotic analgesics for abdominal pain
- •Contraindications to gastric emptying breath test: patients with a known allergy to egg, wheat, or algae.
- •Underlying seizure disorder
- •Known history of cardiac ischemia
- •Recent clinically significant gastrointestinal bleeding
- •Patients taking Levodopa
- •Any other condition, which in the opinion of the investigator would impede compliance or hinder the completion of the study
- •Failure to give informed consent
- •Surgery for placement of a gastric stimulator within the past 6 months (patients > 6 months post-op with persistent symptoms and delayed gastric emptying are eligible).
- •A normal upper endoscopy not performed within 2 year of study entry.
- •Patients taking phosphodiesterase inhibitors such as sildenafil, vardenafil, tadalafil
- •Patients with renal impairment (i.e. Creatinine > 2.5 mg/dL)
- •Patients with hepatic dysfunction (i.e. ALT and AST values > 2.5x ULN and T. bilirubin > 1.5x ULN)
- •Patients with uncontrolled diabetes, such as HbA1c > 10 mg/dl at screening/baseline will be excluded.
研究组 & 干预措施
Sapropterin
open label study of sapropterin dihydrochloride
干预措施: sapropterin dihydrochloride (Drug)
结局指标
主要结局
Gastric Accommodation
时间窗: Baseline, 4 Weeks, 8 Weeks
Gastric Accommodation refers to the reflexive relaxation of the upper stomach after swallowing as measured by the Satiety Test at baseline, 4 weeks, and 8 weeks. Increased gastric accommodation is considered a positive outcome.
次要结局
- Secondary Outcome Measures Will Include Change in Symptom Severity as Measured by the Patient Assessment of Gastrointestinal Disorder-Symptom Severity Index Disorders Symptom Severity Index (PAGI-SYM)(Baseline, 4 Weeks, 8 Weeks)
- Secondary Outcome Measures Will Include Change in Symptom Severity.(Baseline, 4 Weeks, 8 Weeks)
- Secondary Outcome Measures Will Include Change in Quality of Life.(Baseline, 4 Weeks, 8 Weeks)
研究者
Linda Nguyen
Principle Investigator
Stanford University