A Phase 4 Study to Assess Symptoms of Exocrine Pancreatic Insufficiency in Subjects With Cystic Fibrosis or Chronic Pancreatitis Treated With Creon® (Pancrelipase) With an Alternate Source of Active Pharmaceutical Ingredient
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 30
- 试验地点
- 21
- 主要终点
- Mean Change in Total Symptom Score (TSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85
研究概览
简要总结
Exocrine pancreatic insufficiency (EPI) is a condition that is caused by the inadequate pancreatic enzymes needed for normal digestion and is commonly associated with a wide range of chronic diseases, including cystic fibrosis (CF), chronic pancreatitis (CP), and pancreatic cancer. This study will assess clinical symptoms when participants with CF or CP are treated with Creon with alternate source of active drug.
Creon is an approved drug for the treatment of EPI. This study is subject-blinded which means participants will not know the source of the study drug they are given. Approximately 30 adult participants with CF or CP will be enrolled at approximately 15 sites across the Unites States.
Participants will receive oral capsules of CREON for 113 days and will be followed for 30 days.
Participants will attend regular visits during the study at a hospital or clinic or via telemedicine. The effect of the treatment will be checked by medical assessments, checking for side effects and completing questionnaires.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Previous diagnosis of cystic fibrosis (CF) or chronic pancreatitis (CP).
- •Previous diagnosis of exocrine pancreatic insufficiency (EPI) that is currently clinically controlled.
- •Total Symptom Score (TSS) < 1.8 on Pancreatic Exocrine Insufficiency Questionnaire (PEI-Q) at Screening.
排除标准
- •Malignancy involving the digestive tract in the last 5 years, or other significant disease or medical condition that may interfere with EPI symptom assessment.
研究组 & 干预措施
Run-in Period: Creon-ABT
Participants will receive Creon-ABT daily for 28 days.
干预措施: CREON (Drug)
Treatment Period: Creon-AAPIS
Participants will receive Creon-AAPIS daily for 85 days.
干预措施: CREON (Drug)
结局指标
主要结局
Mean Change in Total Symptom Score (TSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85
时间窗: Day 1 (baseline), 8, 15, 29, and 85
The Pancreatic Exocrine Insufficiency Questionnaire (PEI-Q) is an 18-item Patient-Reported Outcome (PRO) instrument that assesses EPI symptoms and associated impact over the previous 7 days. All items are scored using a 5-point Likert scale (Not at all to Yes, a lot). The average score for each symptom domain is calculated, ranging from 0 to 4. TSS is calculated from the average of the mean abdominal domain score and the mean bowel movement symptom score. A numerically higher response indicates more severe EPI symptoms. Positive changes from baseline indicate a worsening of symptoms.
Mean Change in Abdominal Symptom Domain Score (ASDS) From Day 1 (Baseline) to Days 8, 15, 29, and 85
时间窗: Day 1 (Baseline), 8, 15, 29, and 85
The PEI-Q is an 18-item PRO instrument that assesses EPI symptoms and associated impact over the previous 7 days. All items are scored using a 5-point Likert scale (Not at all to Yes, a lot). The average score for each symptom domain is calculated, ranging from 0 to 4. ASDS is the mean abdominal symptom domain score measured by the PEI-Q. A numerically higher response indicates more severe EPI symptoms. Positive changes from baseline indicate a worsening of symptoms.
Mean Change in Bowel Movement Symptom Score (BMSS) From Day 1 (Baseline) to Days 8, 15, 29, and 85
时间窗: Day 1 (Baseline), 8, 15, 29, and 85
The PEI-Q is an 18-item PRO instrument that assesses EPI symptoms and associated impact over the previous 7 days. All items are scored using a 5-point Likert scale (Not at all to Yes, a lot). The average score for each symptom domain is calculated, ranging from 0 to 4. BMSS is the mean bowel movement symptom domain score measured by the PEI-Q. A numerically higher response indicates more severe EPI symptoms. Positive changes from baseline indicate a worsening of symptoms.
次要结局
未报告次要终点