A Phase 1b Study of PTG-100 in Patients With Celiac Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Percentage change in villous height-to crypt ratio
研究概览
简要总结
The goal of this study is to learn whether or not the drug PTG-100 can reduce or prevent inflammatory injury to the small intestine that occurs when people with celiac disease eat food products containing gluten.
This is a clinical research study to determine the safety and efficacy of PTG-100 in preventing gluten-induced inflammatory injury to the small intestine in patients with celiac disease. 30 patients will receive either placebo (fake drug) or PTG-100 (real drug) in capsule form twice daily for 42 days. They will also receive a gluten challenge twice daily in the form of a cookie or equivalent. An upper gastrointestinal endoscopy and exam including small bowel mucosa biopsy will be performed at the start of the treatment period and again at the end. Blood samples will be routinely taken to evaluate safety and the drug's mechanism of action throughout the study, and symptoms will be recorded using the celiac symptoms index (CSI) survey.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
Double-blind
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of celiac disease
排除标准
- •Active GI disease or history of clinically significant diseases
- •Diagnosis of Crohn's disease or ulcerative colitis.
研究组 & 干预措施
PTG-100
Patients will receive either placebo (fake drug) or PTG-100 (real drug) in capsule form twice daily for 42 days.
干预措施: PTG-100 (Drug)
Placebo
Patients will receive either placebo (fake drug) or PTG-100 (real drug) in capsule form twice daily for 42 days.
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage change in villous height-to crypt ratio
时间窗: 42 days
Percentage change from baseline to Week 6 in villous height-to crypt depth (Vh:Cd) ratio
次要结局
- Celiac symptom index (CSI)(42 days)
- Changes in celiac disease antibodies(42 days)
- CD3- positive intraepithelial lymphocyte density(42 days)
- Change in celiac disease antibodies(42 days)
- Characterization of immune modulators underlying mechanism of action in celiac disease.(42 days)
研究者
Nielsen Fernandez-Becker
Clinical Associate Professor, Medicine
Stanford University