Efficacy and Safety of Ultrase MT12 in the Control of Steatorrhea in Cystic Fibrosis (CF) and Pancreatic Insufficient (PI) Children Aged 2 to 6 Years Old
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 49
- 试验地点
- 15
- 主要终点
- Percentage of Patients With Control of Steatorrhea
研究概览
简要总结
Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan).
详细描述
This is a multicenter, explorative, phase IIIb, open-label study in patients with CF and PI. The study consists of a screening visit (visit 1), followed by a baseline phase of 9 days (plus a 5-day window if necessary) during which the regular pancreatic enzyme will be maintained and 10 stool samples will be collected over 5 days, for baseline evaluation of steatorrhea. Afterward, a treatment phase of 19 days (plus a 5-day window if necessary) with Ultrase® MT12 will follow (the usual pancreatic enzyme will be replaced by Ultrase® MT12). Over the last 5 days of the treatment phase, 10 additional stool samples will be collected, for evaluation of steatorrhea.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients aged 2 to 6 years inclusively
- •Patients with current diagnosis of CF based on one or more typical clinical features of CF or a sibling with CF or a positive newborn screening and at least either with sweat chloride test greater than or equal to 60 millimoles/liter (mmol/L) by quantitative pilocarpine iontophoresis on two separate occasions or two identifiable CF-causing mutations
- •Patients with presence of PI as demonstrated by fecal elastase (FE-1) less than 100 microgram/gram (mcg/g) of stools (performed by ScheBo test) and requiring pancreatic enzyme supplementation
- •Patients who are able to eat a high-fat diet calculated at a value between 2g to 4g fat/kg of body weight per day during the whole study and having a current adequate nutritional status based on the body mass index (BMI) greater than or equal to fifth percentile
- •Patients receiving current treatment of PI with pancreatic enzymes
- •The parent or legal guardian signed informed consent form (ICF) and is mentally able to understand and comply with the study procedures
排除标准
- •Patients currently receiving or received an Ultrase® MT product (MT12, MT18, MT20) for PI in the last 30 days
- •Patients having known contraindication, sensitivity or hypersensitivity to Ultrase® or to any porcine protein
- •Patients with presence of a medical condition known to increase fecal fat loss or that could compromise study results or the study patient safety
- •Patients with current diagnosis or history of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months or who had 2 or more episodes of incomplete DIOS in the past year
- •Patients with use of any prohibited medication or product at study entry and during the course of the study
- •Patients with chronic use of narcotics
- •Patients with use of bowel stimulants and/or laxatives more than once a week
- •Patients with presence of acute pancreatitis or exacerbation of chronic pancreatic disease
- •Patients with presence of an acute infection that needed to be treated with oral or intravenous (IV) broad-spectrum antibiotics
- •Patients having history of significant bowel resection; small bowel resection for meconium ileus at birth and appendectomy were accepted. Patients with Presence of dysmotility disorders
- •Patients with presence of chronic or severe abdominal pain
- •Patients unable to comply with diet requirement
- •Patients receiving enteral tube feeding overnight at study entry or who will need to receive enteral tube feeding overnight during the course of the study
- •Patients with history of or a current diagnosis of clinically significant portal hypertension
- •Patients with presence of poorly controlled diabetes according to the Investigator's clinical judgment
- •Patients having any condition or pre-study laboratory abnormality or history of any illness which, in the opinion of the Investigator, might have put the patient at risk, prevented the patient from completing the study, or otherwise affect the outcome of the study
- •Patient with use of any investigational drug within 30 days prior to the date of signature of the ICF
研究组 & 干预措施
Ultrase® MT12
干预措施: Ultrase® MT12 (Drug)
结局指标
主要结局
Percentage of Patients With Control of Steatorrhea
时间窗: A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)
Control of steatorrhea was defined as a less than 30 percent (%) of fat in stools as measured by nuclear magnetic resonance (NMR) spectroscopy in all stool samples which are collected at baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and during the 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12.
次要结局
- Percentage of Stools With Abnormal Characteristics(A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3))
- Percentage of Patients With Normal Stool Frequency(A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3))
- Percentage of Stools With Normal Consistency(A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3))
- Mean Number of Days Without Abdominal Complaints(A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3))