A Phase 1 Open-Label, Multicenter Study to Assess the Safety and Efficacy of ANG003 in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 51
- 试验地点
- 20
- 主要终点
- Adverse Events (AE), Serious Adverse Events (SAE), and AEs Leading to Study Discontinuation
研究概览
简要总结
Randomized, parallel, active-treatment Phase 1 study of a single dose of orally administered ANG003 with a test meal in adult subjects with cystic fibrosis-related exocrine pancreatic insufficiency. The study's overall objectives are to evaluate the safety, tolerability and effect of four dose levels of ANG003.
详细描述
The Phase 1 study was designed to compare a 24h Baseline Substrate Absorption Challenge Test (SACT) period with no enzymes to a 24h ANG003 SACT period with enzymes. Eligible subjects will be randomly assigned with equal allocation to one of four active dose levels of lipase, protease and amylase. Approximately 48 to 60 eligible subjects are planned to be enrolled in the study with 12 to 15 subjects assigned to each dose level from up to 21 investigational sites.
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
- None
盲法说明
Open-label
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
- •Male and female subjects 18 years of age or older.
- •Confirmed diagnosis of CF defined as: a) CF signs and symptoms AND b) Two CF-causing mutations on genetic testing or sweat chloride >60 mEq/L.
- •Documented history of fecal elastase <100 µg/g stool.
- •EPI clinically controlled with minimal clinical symptoms and on a stable dose of PERT for 90 days before screening as determined by the Investigator.
- •Adequate nutritional status measured by body mass index ≥20kg/m2 for adult subjects.
排除标准
- •Subjects with diabetes mellitus who are unable to refrain from short-acting and rapid-acting insulin on Days 1 and 5 for a daily total of 6 hours.
- •Involuntary loss of ≥10% of usual body weight within last 6 months or involuntary loss of >5% of body weight within 1 month.
- •Requires use of naso-gastric, J-tube, G-tube, and/or enteral feeding for the study duration.
- •CF pulmonary exacerbation within 30 days prior to the Baseline SACT Period (Visit 2).
- •Subjects who cannot discontinue omega-3 supplements >500 mg of DHA and EPA daily.
- •Subjects unable to tolerate missing a dose of PERT.
研究组 & 干预措施
ANG003 Dose Level 1
Single administration starting dose contains lipase, protease and amylase.
干预措施: ANG003 (Drug)
ANG003 Dose Level 2
Single administration dose contains 2x lipase, 2x protease and 2x amylase starting dose.
干预措施: ANG003 (Drug)
ANG003 Dose Level 3
Single administration dose contains 4x lipase, 2x protease and 2x amylase starting dose.
干预措施: ANG003 (Drug)
ANG003 Dose Level 4
Single administration dose contains 6x lipase, 3x protease and 3x amylase starting dose.
干预措施: ANG003 (Drug)
结局指标
主要结局
Adverse Events (AE), Serious Adverse Events (SAE), and AEs Leading to Study Discontinuation
时间窗: Assessed through study completion, up to 9 days (Day 1 thru Day 9).
AE, SAE, and AEs leading to study discontinuation measured by frequency and severity during study.
Malabsorption Symptoms
时间窗: Acute PAGI-SYM is based upon 7-day recall.
Severity of malabsorption symptoms (e.g., abdominal discomfort or pain, bloating, heartburn, regurgitation or reflux, retching, stomach fullness, and vomiting) by patient reported outcome scale (0=None; 1=Very Mild; 2=Mild; 3=Moderate; 4=Severe; 5=Very Severe). Measured by 20 questions contained in Acute Patient Assessment of Gastrointestinal Symptoms (Acute PAGI-SYM) questionnaire.
次要结局
- Fat Absorption(Assessed through study completion, up to 9 days (Day 1 thru Day 9).)
- Protein Absorption(Assessed through study completion, up to 9 days (Day 1 thru Day 9).)
- Carbohydrate Absorption(Assessed through study completion, up to 9 days (Day 1 thru Day 9).)