OPTION 2: A Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to Assess the Safety and Efficacy of MS1819 in Enteric Capsules in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis; With an Extension Phase Evaluation of Immediate Release MS1819 Capsules
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 15
- 主要终点
- Efficacy of MS1819: Coefficient of Fat Absorption (CFA)
研究概览
简要总结
The primary objectives of this study are to assess the safety and efficacy of MS1819 in enteric capsules vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). The exploratory objective of the extension phase (EP) is to find a dose of MS1819 in immediate release capsules that is safe and results in CFA values in a therapeutic range.
详细描述
This is a Phase 2, open-label, multicenter, 2, 2x2 crossover study assessing the safety and efficacy of MS1819, 2240 mg/day vs porcine PERT, and 4480 mg/day vs porcine PERT given at the same dose and dosing regimen that was being administered during the pre-study period. MS1819 will be administered in enteric capsules.
MS1819 will be assessed in a 2x2 crossover including approximately 30 patients completing both periods. Fifteen patients will be randomized to the MS1819 2240 mg/day vs PERT arm, and 15 patients will be randomized to the MS1819 4480 mg/day vs PERT arm. Patients in each arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.The coefficient of fat absorption (CFA) will be measured at the end of each 3 week study period. Patients enrolled into the extension phase (EP) will be composed of patients who have completed the crossover phase of OPTION 2.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
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
- •Cystic fibrosis, based on 2 clinical features consistent with CF, plus either a new/historic sweat chloride ≥60 mmol/L (measured while not on a CFTR modulator) or genotype.
- •Under stable dose of porcine PERT
- •A fair or better nutritional status
- •Fecal elastase <100 µg/g
- •Standard-of-care medications including CFTR modulators are allowed
排除标准
- •History or diagnosis of fibrosing colonopathy
- •Any chronic diarrheal illness unrelated to pancreatic insufficiency
- •Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level ≥5 ×upper limit of normal (ULN), or total bilirubin level ≥1.5 ×ULN at the Screening visit
- •Feeding via an enteral tube during 6 months before screening
- •Forced expiratory volume ≤30% at the Screening visit
研究组 & 干预措施
MS1819 2240 mg/day vs PERT arm,
Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.
干预措施: MS1819 (Drug)
MS1819 2240 mg/day vs PERT arm,
Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.
干预措施: Porcine PERT (Drug)
MS1819 4480 mg/day vs PERT arm
Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.
干预措施: MS1819 (Drug)
MS1819 4480 mg/day vs PERT arm
Patients in arm will further be randomized to receive either the sequence consisting of MS1819 for 3 weeks followed by PERT for another 3 weeks or the opposite sequence of treatments, PERT for 3 weeks followed by MS1819 for another 3 weeks.
干预措施: Porcine PERT (Drug)
结局指标
主要结局
Efficacy of MS1819: Coefficient of Fat Absorption (CFA)
时间窗: 6 weeks for crossover phase (to complete both first and second intervention) and 2 weeks for extension phase.
The primary efficacy endpoint is the CFA that will be assessed at the end of each 3-week treatment period (crossover period) and 2-week treatment period (Extension period). CFAs for MS1819 will be compared to the CFAs of PERT using descriptive methods.
Safety of MS1819 by Number of Subjects Reporting 1 or More Adverse Events
时间窗: Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period)
Number of subjects reporting 1 or more adverse events
次要结局
- Stool Weights(Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period))
- Coefficient of Nitrogen Absorption (CNA)(Each 3-week treatment period (crossover period) and 2-week treatment period (Extension period))