EUCTR2017-000571-85-HU进行中（未招募）1 期
A Phase 3, Randomized, Open-Label, Assessor-Blind, Non-Inferiority, Active-Comparator Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency - RESULT: Reliable, Emergent Solution Using Liprotamase Treatment
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 192
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1.Cystic Fibrosis based on the disease diagnostic criteria:
- •Two clinical features consistent with CF, and
- •a.genotype with 2 identifiable mutations consistent with CF, or
- •b.sweat chloride = 60 mEq/L (=60 mmol/L) by quantitative pilocarpine iontophoresis.
- •Diagnostic evidence of CF disease may be measured during screening unless previously documented.
- •2.Age = 7 years at the time of screening.
- •3.Fecal elastase <100 mcg/g stool measured during screening.
- •4.CFA during screening of =80% while receiving PERT therapy. Subjects who meet all enrollment criteria except CFA may be re screened for CFA after an increase in pre-enrollment PERT dose for at least 1 week if this is deemed to be safe and appropriate by the Investigator. Such rescreening of CFA should occur within 30 days of the result of the first CFA measurement.
- •5.Clinically stable with no evidence of acute upper or lower respiratory tract infection in the last 10 days prior to Visit 1.
- •6.Fair or better nutritional status as defined by:
- •BMI =16.0 kg/m2 for female subjects =18 years of age, or
- •BMI =16.5 kg/m2 for male subjects =18 years of age, or
- •BMI =25th percentile for subjects 7 to <18 years of age based on growth charts published by the World Health Organization (WHO).
- •Weight for all age groups has remained stable (no more than a 5% decline) during the 90 days prior to screening.
- •7.Able to take pancreatic enzyme supplementation in the form of capsules.
- •8.Using the same pancreatic enzyme treatment for the past 30 days without change in dosing regimen unless the change in regimen occurs during CFA rescreening as described in Inclusion Criterion #4.
- •9.Subjects receiving gastric acid suppression therapy (proton pump inhibitor or histamine 2 antagonist) must have been on a stable dose for at least 30 days prior to Visit 2.
- •10.Able to perform testing and procedures required for the study, as judged by the Investigator.
- •11.If adult, have given written informed consent or assent as required by Anthera or its designee and the Institutional Review Board/Independent Ethic Committee (IRB/IEC). If not adult, have parent or legal guardian who has given written informed consent approved by Anthera or its designee and the IRB/IEC governing the site.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 70
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 80
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1.Presently using Pancreaze as enzyme preparation for treatment of EPI. Patients are not required to be Pancreaze naïve but should not have received Pancreaze within 30 days of screening.
- •2.Treatment with liprotamase in a previous clinical trial including Study TC-2A, Study 726, Study 767, the SIMPLICITY study (AN-EPI3332), and the SOLUTION trial (AN EPI3331).
- •3.Women who are breast-feeding during the study.
- •4.A history or diagnosis of fibrosing colonopathy (FC).
- •5.History of distal intestinal obstruction syndrome (DIOS) in the 6 months prior to the screening visit.
- •6.Any chronic diarrheal illness unrelated to pancreatic insufficiency (e.g., infectious gastroenteritis, sprue or inflammatory bowel disease).
- •7.A history of solid organ transplant, or significant surgical resection of the bowel. Significant resection of the bowel is defined as any resection of the terminal ileum or ileocecal valve. Subjects who have had qualitative, long term changes in nutritional status after any other bowel resection (e.g., increased or new need for pancreatic enzyme supplementation compared to pre operative status in order to maintain the same nutritional status) should also be excluded.
- •8.Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level =5 times upper limit of normal (ULN), or total bilirubin level =1.5 times ULN at the screening visit, unless due to Gilbert’s syndrome. Cases of suspected or confirmed Gilbert’s syndrome should be discussed with the Medical Monitor.
- •9.Signs and/or symptoms of liver cirrhosis or portal hypertension (e.g., splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF.
- •10.Forced expiratory volume in 1 second (FEV1) <30% of the predicted value.
- •11.Use of an enzyme preparation at screening in excess of 10,000 U lipase/kg/day or 2,500 U lipase/kg per meal in patients =12 years of age, or 6,700 U lipase/kg/day in patients <12 years of age.
- •12.Known hypersensitivity to food additives or Pancreaze.
- •13.Uncontrolled hyperglycemia or uncontrolled CF-related diabetes.
- •14.Uncontrolled hyperuricemia or hyperuricosuria as determined by the principal Investigator (PI).
- •15.Feeding via an enteral tube during 6 months prior to screening.
- •16.Routine use of anti-diarrheals, anti-spasmodics, or cathartic laxatives, or a change in chronic osmotic laxative (e.g., polyethylene glycol) regimen in the previous 3 months.
- •17.Any condition that the Investigator believes would interfere with the intent of this study or would make participation not in the best interests of the subject.
- •18.Unlikely to complete the study, as determined by the Investigator.
- •19.Currently enrolled in, or have discontinued within the last 30 days (prior to the screening visit) from a clinical trial involving use of an investigational drug, biologic or device, or are currently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study.
- •20. Female who is nursing, pregnant, intending to become pregnant or intending to nurse during the time of the study, or who have a positive pregnancy test at baseline. All sexually-active subjects of reproductive potential are required to use a reliable method of birth control. Females and males are required to use or start using a reliable method of birth control at least 2 weeks prior to randomization, throughout the study, and for at least 3 months following completion of study ther
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