A Multicenter, Randomized, Open-Label, Crossover Study to Evaluate the Mode of Administration and Safety of EUR-1008 in Infants 1 to 12 Months of Age With Exocrine Pancreatic Insufficiency (EPI) Associated With Cystic Fibrosis (CF)
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 15
- 试验地点
- 6
- 主要终点
- Question 6 (Previous Pancreatic Enzyme Product [PEP])
研究概览
简要总结
A study to determine the safety, effectiveness, and acceptability of 2 methods of administration of EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000 lipase units capsule, a pancreatic enzyme product (PEP), in infants with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). This study is sponsored by Aptalis Pharma (formerly Eurand).
详细描述
This is a multicenter, randomized, open-label, crossover study in pediatric participants with EPI due to CF. The study will be carried out in infants between 1 and 12 months of age.
The study comprises of a screening period (up to 10 days) followed by 2 treatment periods (10 days each). During the screening period, all participants will be administered Zenpep® 5,000 (pancrelipase) mixed with a small amount of apple sauce. Once determined eligible for participation, participants will be randomized into 1 of 2 treatment sequences. Each sequence corresponds to taking one treatment in the first period and the other treatment in the second period, and were administered EUR-1008 (APT-1008) (Zenpep® [pancrelipase] delayed release capsule) 3,000 lipase units capsule either mixed with apple juice using a syringe nurser or apple sauce using a spoon.
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
- 1 Month 至 12 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with diagnosis of CF based on the following criteria: one clinical feature consistent with CF, and either a genotype with 2 identifiable mutations known to cause CF or a sweat chloride concentration that is greater than 60 milliequivalent per liter by quantitative pilocarpine iontophoresis
- •Participants who have the need for a PEP defined as monoclonal fecal elastase less than 200 microgram per gram (mcg/g) stool
- •Caregiver must be willing to switch participant from their previous PEP (if any) to Zenpep®
- •Participants who have a height to weight ratio target at greater than tenth percentile
- •Participants who are clinically stable with no evidence of concomitant illness or acute upper or lower respiratory tract infection during the 7-day interval prior to screening and preceding accession into this clinical study
排除标准
- •Participants who are less than 1 month old or are greater than 12 months old
- •Participants with history of meconium ileus or small bowel atresia in the newborn period that required surgery
- •Participants who are allergic to pork or other porcine PEPs
- •Participants with any respiratory condition or other serious comorbidity (for example patent ductus arteriosus [PDA], or necrotizing enterocolitis [NEC]) that in the investigator's opinion would result in an intervention requiring hospitalization or intensive pulmonary or other treatment during the trial
- •Participants with other comorbidities independent of CF that, in the investigator's opinion, would result in an inability to participate in the study or excess risk to the participant that is above the standard of care
- •Participants with acute respiratory infection in the previous 14 days requiring antibiotics
- •Participants who required change in antacid dose in the 7 days before screening
- •Participants with administration of oral, intramuscular (IM), intravenous (IV) glucocorticoids in the 4 weeks prior to screening
- •Participants with any condition that would, in the investigator's opinion, limit the participant's ability to complete the study
- •Participants currently participating in or has participated in an investigational study, with the exception of observational studies, within 30 days of the screening visit
研究组 & 干预措施
EUR-1008 (APT-1008) in Apple Juice
干预措施: EUR-1008 (APT-1008) (Drug)
EUR-1008 (APT-1008) in Apple Sauce
干预措施: EUR-1008 (APT-1008) (Drug)
结局指标
主要结局
Question 6 (Previous Pancreatic Enzyme Product [PEP])
时间窗: Baseline
Acceptability questionnaire consists of 9 questions (Q) to assess the ease, time, overall satisfaction of study drug. Q6 included "name of previous PEP administered". Q6 was reported as number of participants who used any PEP prior to screening.
Treatment Difference for Acceptability of Treatment
时间窗: Baseline up to end of study (Day 21)
Acceptability questionnaire consists of 9 question (Q) to assess ease, time, overall satisfaction of study drug. Rated on 5-point scale for Q1-Q5 and Q7-Q9; Q6 was not rated and asked for name of previous PEP administered. Q1=overall ease of administration (1=not at all easy,2=somewhat,3=easy,4=very,5=extremely); Q2=time of administration (1=very short\[\<2 min\],2=short\[2-5 min\],3=moderate\[5-15 min\],4=long\[15-25 min\],5=very long\[\>25 min\]);Q3=overall infant acceptance(1=very easily,2=easily,3=same,4=with difficulty,5=with great difficulty);Q4=clear/complete instructions(1=not clear,2=somewhat,3=clear,4=very,5=extremely);Q5=overall satisfaction with dosing method (1=not satisfied,2=somewhat,3=satisfied,4=very,5=extremely);Q7=comparative ease of administration (1=much worse,2=worse,3=same,4=better,5=much better);Q8=comparative infant acceptance (1=much more difficult,2=more,3=same,4=easier,5=much easier);Q9=comparative overall satisfaction (1=much less,2=less,3=same,4=more,5=much more).
次要结局
- Number of Abdominal Symptoms: Bloating(Up to Day 10 in first and second treatment periods, end of study (Day 21))
- Number of Stools Categorized as Per Consistency(Up to Day 10 in first and second treatment periods, end of study (Day 21))
- Number of Abdominal Symptoms: Flatulence(Up to Day 10 in first and second treatment periods, end of study (Day 21))
- Number of Participants With Abnormal Clinical Laboratory and Vital Signs Findings(Baseline up to end of study (Day 21))
- Number of Participants With Abnormal Findings With Respect to Oral Mucosa(Baseline up to end of study (Day 21))
- Daily Number of Stools(Up to Day 10 in first and second treatment periods, end of study (Day 21))
- Number of Stools With Signs of Blood and Visible Oil or Grease(Up to Day 10 in first and second treatment periods, end of study (Day 21))
- Number of Abdominal Pain Symptoms(Up to Day 10 in first and second treatment periods, end of study (Day 21))