A Phase 2, Multicenter, Randomized, Active-controlled Study to Assess the Safety and Efficacy of ANG003 at Two Different Dose Levels in Subjects With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 113
- 试验地点
- 32
- 主要终点
- Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values
研究概览
简要总结
In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine pancreatic insufficiency (EPI). People with EPI due to Cystic Fibrosis (CF) may be eligible to participate in this study. The primary objective of this study is to evaluate the safety of ANG003 and see if it works as well compared to Creon, an approved PERT.
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
- Single (Outcomes Assessor)
盲法说明
A centralized, blinded safety assessor will be responsible for conducting independent reviews of key safety data to ensure objective and consistent evaluation across all study sites.
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female subjects aged ≥12 years from the date of informed consent in the US and aged ≥18 years in the EU
- •Confirmed and documented diagnosis of CF
- •Diagnosed with severe EPI as defined by a fecal elastase ≤50 μg/g stool measured at Screening by a central laboratory.
- •Subject has controlled EPI and taking a stable dose of pancreatic enzyme replacement therapy (PERT) for 90 days prior to Screening.
- •Adequate nutritional status measured by body mass index (BMI) defined by:
- •BMI ≥25th percentile for children aged 12-17 years
- •BMI ≥18.5 kg/m2 for ≥18 years of age
排除标准
- •History of fibrosing colonopathy or recurring distal intestinal obstructive syndrome within 6 months of Screening.
- •History of lung or liver transplant, listing for organ transplant or significant bowel resection within the last 6 months. Subjects with a history of resection that does not result in short bowel syndrome are eligible.
- •Known hypersensitivity or other severe reaction to any ingredient of the investigational product (IP), Creon, or the stool marker (FD&C Blue #2).
- •Any chronic diarrheal illness unrelated to pancreatic insufficiency, actively being treated for small intestinal bacterial overgrowth, requiring use of naso-gastric, J-tube, G-tube, and/or enteral feeding for the study duration, Clostridioides difficile infection within 6 months prior to Screening, or severe constipation defined as <1 bowel movement/week.
研究组 & 干预措施
60% to <80% CFA ANG003 Dose B
All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. Participants with a Period A CFA result of 60% to <80% will be assigned to ANG003 Dose B.
干预措施: ANG003 Dose B (Drug)
ANG003 Dose A
All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to ANG003 Dose A for a 21-day acclimation and subsequent CFA analysis.
干预措施: ANG003 Dose A (Drug)
Off Enzyme
All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to the off enzyme arm. Subjects will receive Creon for an additional 21-day and the subsequent CFA analysis will be off enzyme. Total time off enzyme will be 4-7 days.
ANG003 Dose B
All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to ANG003 Dose B for a 21-day acclimation and subsequent CFA analysis.
干预措施: ANG003 Dose B (Drug)
Creon
All subjects will be acclimated to Creon for 14-days and then will undergo a baseline CFA analysis. If baseline CFA is >/= 80%, the subject may be randomized to Creon for an additional 21-days and subsequent CFA analysis.
干预措施: Creon (Drug)
结局指标
主要结局
Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values
时间窗: From Visit 1 to Visit 5, anticipated average 80 days
Adverse events (AEs); Treatment-emergent AEs; Serious AEs; Discontinuation due to AEs; Clinical laboratory values
Coefficient of Fat Absorption (CFA)
时间窗: Period A (baseline) and Period B 72 hour CFA collection
Mean change from baseline CFA (i.e., Period B CFA minus Period A CFA) assessed for ANG003 Dose A, ANG003 Dose B, and Creon amongst subjects with baseline CFA ≥80%.
次要结局
- Plasma docosahexaenoic acid (DHA) and eicosapentaenoic acid (EPA) (ug/mL)(Period A (baseline) and Period B time 0, 1, 2, 4, 6, 8, 10 and 24 hour collections)
- Coefficient of Nitrogen Absorption (CNA)(Period A (baseline) and Period B 72 hour CNA collection)
- % of subjects with GI symptoms(From Visit 2 to Visit 5, anticipated average 59 days)
- Cumulative stool weight(Period A (baseline) and Period B 72 hour collection)
- Number of Stools per Day(Period A (baseline) and Period B 72 hour collection)