A Phase 2, Open-Label, Multicenter, 2x2 Crossover Trial to Assess the Safety and Efficacy of MS1819-SD in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 41
- 试验地点
- 14
- 主要终点
- Efficacy of MS1819-SD: 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-SD vs porcine pancreatic enzyme replacement therapy (PERT) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF).
详细描述
This is a Phase 2, open-label, multi-center, 2x2 crossover study assessing the safety and efficacy of MS1819-SD (spray dried) vs porcine PERT given at the same dose that was being administered during the pre-study period.
MS1819-SD will be assessed in a 2x2 crossover including at least 30 patients completing both periods.
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
盲法说明
Unblinded
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 initial diagnostic sweat chloride ≥ 60 mmol/L
- •Under stable dose of porcine PERT
- •A fair or better nutritional status
- •Fecal elastase <100 µg/g
- •Standard-of-care medications including Cystic Fibrosis Transmembrane Conductance Regulator (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 (3 weeks) then PERT pre-study dose (3 weeks)
Patients in arm will further be randomized to receive either the sequence consisting of MS1819 2240 mg/day for 3 weeks followed by PERT for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose.
干预措施: MS1819 (Drug)
MS1819 2240 mg/day (3 weeks) then PERT pre-study dose (3 weeks)
Patients in arm will further be randomized to receive either the sequence consisting of MS1819 2240 mg/day for 3 weeks followed by PERT for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose.
干预措施: Porcine PERT (Drug)
PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks)
Patients in arm will be randomized to receive PERT for 3 weeks followed by MS1819 2240 mg/day for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose.
干预措施: MS1819 (Drug)
PERT pre-study dose(3 weeks) then MS1819 2240 mg/day (3 weeks)
Patients in arm will be randomized to receive PERT for 3 weeks followed by MS1819 2240 mg/day for another 3 weeks. During the PERT treatment period the patients will take their stable pre-study PERT dose.
干预措施: Porcine PERT (Drug)
结局指标
主要结局
Efficacy of MS1819-SD: Coefficient of Fat Absorption (CFA)
时间窗: 3 weeks
The Coefficient of Fat Absorption (CFA%) is defined as: \[72-hour fat intake (g) - 72-hour fat excretion (g)/72-hour fat intake(g)\] x 100 = CFA% The threshold for CFA results (\>80%) is considered clinically significant for treatment effectiveness by the FDA.
Safety of MS1819-SD by Number of Participants Reporting 1 or More Adverse Events (AE)
时间窗: 6 weeks
Number of participants reporting 1 or more adverse events
Safety of MS1819-SD by Number of Treatment Emergent Adverse Events (TEAEs)
时间窗: 6 weeks
Number of Treatment emergent adverse events
次要结局
- Stool Weights(6 weeks)
- Signs and Symptoms of Malabsorption(3 weeks)
- Coefficient of Nitrogen Absorption (CNA)(3 weeks per group.)