NCT00981214已完成3 期
An Open-Label Study to Evaluate the Efficacy and Safety of Pancreatic Enzyme Product (PEP) Microtabs in Pediatric Patients With Cystic Fibrosis and Exocrine Pancreatic Insufficiency
Forest Laboratories14 个研究点 分布在 1 个国家目标入组 19 人开始时间: 2006年5月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 19
- 试验地点
- 14
- 主要终点
- Percentage of Participants Who Were Responders After 1 Week of Treatment With Study Medication
研究概览
简要总结
This is an open-label study to evaluate the efficacy and safety of Aptalis' (formerly Eurand) pancreatic enzyme product (PEP) microtabs in pediatric participants under age 7 with cystic fibrosis (CF) and exocrine pancreatic insufficiency (EPI).
详细描述
The study sample will consist of evaluable participants, all of whom will be children younger than 7 years of age. Participants will receive EUR-1008 (APT-1008) Microtabs formulation. The study design involves a 4-day screening period, a 7-day dose stabilization period, and a 7-day treatment period (excluding an end-of-study evaluation).
The optimal dose of EUR-1008 (APT-1008) Microtabs, determined during the dose stabilization period, will be used during the treatment period. Participants are instructed to consume a predefined diet.
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
- — 至 7 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants less than 7 years of age
- •Participants who have pancreatic insufficiency documented by a fecal elastase level less than 100 micrograms per gram (mcg/g), or if not documented, the fecal elastase test must be done at the screening visit
- •Participants who have a need of de novo treatment with pancreatic enzymes or be able to be switched from an existing treatment
- •Participants who have a body mass index greater than the twenty fifth percentile for children 2 years and older
- •Participants with a weight for height index greater than the twenty fifth percentile for children less than 2 years of age
- •Participants with diagnosis of CF based upon the following criteria:
- •Have 2 clinical features consistent with CF and
- •Have either a genotype with 2 identifiable mutations consistent with CF or a sweat chloride concentration that is more than 60 milliequivalent per liter (mEq/L) by quantitative pilocarpine iontophoresis
- •Participants who are clinically stable with no evidence of acute upper or lower respiratory tract infection
排除标准
- •Participants with fibrosing colonopathy
- •Participants allergic to pork or other porcine PEPs
- •Participants with any respiratory condition that in the investigator's opinion would result in an intervention requiring hospitalization or intensive pulmonary treatment during the trial
- •Participants with any acute systemic administration of an antibiotic for any reason in the previous 4 weeks; however, a low stable dose of an antibiotic (such as azithromycin 250 or 500 milligram [mg] up to 3 times per week) is allowed. Moreover, chronic treatment (that is, daily for at least 1 month) with an inhalatory antibiotic (for example, colistin, tobramycin, or ceftazidime) is allowed
- •Participants who have hepatic insufficiency as defined by a history or presence of ascites, or a serum albumin level of less than 3.0 milligram per deciliter (mg/dL), or coagulopathy with an international normalized ratio that is greater than 1.7
- •Participants with hyperuricemia or hyperuricosuria
- •Participants participating in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to screening visit
- •Participants with history of or current screening evaluation of hyperglycemia as defined by an 8-hour fasting serum glucose equivalent to 126 mg/dL or more, or of cystic-fibrosis-related diabetes as determined according to the Cystic Fibrosis Foundation (CFF) Consensus Conference of January 1999 (Section IX Part II), that is:
- •Fasting Blood Glucose (FBG) greater than126 mg/dl (7.0 milli mole [mM]) on two or more occasions
- •FBG greater than 126 mg/dl (7 .0 mM) plus casual (without regard to time of day or last meal consumed) glucose level greater than200 mg/dl (11.1 mM)
- •Casual (previously called random) glucose levels greater than 200 mg/dl (11.1 mM) on two or more occasions with symptoms
- •Participants with any solid organ transplant or surgery affecting the bowel
- •Participants using an enzyme preparation in excess of 10,000 lipase units/kg/day
- •Participants with an acute dose of any steroid in the previous 2 weeks; however, low chronic doses of a steroid (less 0.5 mg/kg every other day) will be allowed
- •Participants with any condition that would, in the investigator's opinion, limit the patient's ability to complete the study
- •Participants with history of or current screening determination of distal ileal obstruction syndrome (DIOS), or any clinical signs and symptoms suggestive of DIOS (that is, constipation, abdominal pain, anorexia, early satiety, recurrent vomiting and palpable fecal mass) on physical examination
- •Participants who are unable to discontinue excluded concomitant medications over the course of the study
研究组 & 干预措施
EUR-1008 (APT-1008)
Experimental
干预措施: EUR-1008 (APT-1008) (Drug)
结局指标
主要结局
Percentage of Participants Who Were Responders After 1 Week of Treatment With Study Medication
时间窗: Day 11
Responders were defined as those participants without steatorrhea (defined as less than 30 percent (%) fecal fat content) and without signs and symptoms of malabsorption after 1 week of treatment with study medication.
Percentage of Participants Who Were Responders After 2 Weeks of Treatment With Study Medication
时间窗: Day 18 (end of treatment)
Responders were defined as those participants without steatorrhea (defined as less than 30% fecal fat content) and without signs and symptoms of malabsorption after 2 weeks of treatment with study medication.
次要结局
- Change From Baseline in Weight at Day 12, 19(Baseline, Day 12, 19)
- Mean Daily Number of Stools(Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period))
- Percentage of Blood in Stool(Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period))
- Percentage of Stool Categorized by Consistency(Baseline, Day 5 up to Day 11 (dose stabilization period) and Day 12 up to Day 18 (treatment period))
- Physician's and Parent's or Legal Guardians Assessment of Improvement in Clinical Symptoms(Day 19 (end of study))
- Mean Number of Abdominal Symptoms: Bloating(Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period))
- Mean Number of Abdominal Symptoms: Flatulence(Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period))
- Mean Number of Pain Symptoms(Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period))
- Percentage of Stool With Visible Oil or Grease(Baseline, Day 5 up to Day 11 (dose stabilization period), Day 12 up to Day 18 (treatment period))
研究者
研究点 (14)
Loading locations...
相似试验
已完成
2 期
An Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (ACROBAT Edge)AcromegalyNCT03789656Crinetics Pharmaceuticals Inc.47
已完成
2 期
Study to Assess the Safety, Tolerability, Efficacy and PK of APL-2 in Patients With Warm Type Autoimmune Hemolytic Anemia (wAIHA) or Cold Agglutinin Disease (CAD)Warm Autoimmune Hemolytic AnemiaCold Agglutinin DiseaseNCT03226678Apellis Pharmaceuticals, Inc.24
终止
不适用
A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With DermatomyositisDermatomyositisNCT01140503Stanford University5
已完成
1 期
Study of Adavosertib(AZD1775) in Japanese Patients With Advanced Solid TumoursAdvanced Solid TumoursNCT04462952AstraZeneca6
已完成
3 期
A Study to Evaluate the Safety of Raptiva in Adults With Plaque Psoriasis Previously Enrolled in Study ACD2600gPsoriasisNCT00096603Genentech, Inc.450