Randomized, Double-blind, Cross-over Trial Assessing a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 2
- 主要终点
- Maximum serum triglyceride concentration
研究概览
简要总结
This is a single center, randomized, double-blind, cross-over trial (with a follow-on single-blind safety evaluation stage), assessing a ready-to-drink nutritional supplement used without PERT ("PERT-free"), nutritional supplement for blood lipid levels, safety and tolerability compared to a standard of care nutritional supplement used concomitantly with PERT.
详细描述
This is a single center, randomized, double-blind, cross-over trial (with a follow-on single-blind safety evaluation stage), assessing a ready-to-drink nutritional supplement used without PERT ("PERT-free"), nutritional supplement for blood lipid levels, safety and tolerability compared to a standard of care nutritional supplement used concomitantly with PERT.
The study hypothesis is that the PERT-free nutritional supplement will be equivalent to, or superior to, a standardized nutritional shake with regard to fat absorption, as indicated by blood lipid (triglyceride) levels while maintaining tolerability and safety without the use of PERT. The objective is to evaluate efficacy, safety, and tolerability of a "PERT-free" nutritional shake compared to a standard nutritional shake used concomitantly with PERT. Measurements include components of a standard lipid blood panel (triglycerides, cholesterol, HDL cholesterol, LDL cholesterol, and VLDL cholesterol), absorption of fat-soluble vitamins (vitamin D) and symptoms associated with EPI. Other objectives include evaluating palatability and patients' impressions of the products.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Provision of signed and dated informed consent form.
- •Stated willingness to comply with all study procedures and availability for the duration of the study.
- •Male or female, aged 12 years or older.
- •Diagnosis of cystic fibrosis
- •Currently receiving treatment with a commercially available pancreatic enzyme product for more than 3 months.
- •Clinically stable condition without evidence of acute respiratory disease within 1 month of enrollment.
- •Stable body weight defined as no more than 5% decline within 3 months of enrollment.
- •Females of child-bearing potential should agree to continue using a medically acceptable method of birth control throughout the study and for 30 days immediately after the last dose of study drug. Medically acceptable methods of birth control include bilateral tubal ligation or the use of either a contraceptive implant, a contraceptive injection (Depo-Provera™), an intrauterine device, or an oral contraceptive taken within the past 3 months where the subject agrees to continue using during the study or to adopt another birth control method, or a double-barrier method which consists of a combination of any two of the following: diaphragm, cervical cap, condom, or spermicide.
- •Ability to take oral medication and oral liquid nutritional supplements and be willing to adhere to the study interventions.
- •Agreement to adhere to Lifestyle Considerations as defined by the study protocol throughout the duration of the study.
排除标准
- •Evidence of cardiovascular, respiratory (except underlying disease), urogenital, gastrointestinal/hepatic (except underlying disease), hematologic/immunologic, head, ears, eyes, nose, throat, dermatologic/connective tissue, musculoskeletal, metabolic/nutritional (except underlying disease), endocrine (except controlled diabetes mellitus), neurologic/psychiatric, milk, nut or soy allergies, recent major surgery, or other relevant diseases as revealed by history, physical examination and/or laboratory assessments, which could limit participation in or completion of the study.
- •History of acute abdomen in the last year.
- •History of fibrosing colonopathy.
- •History of distal intestinal obstruction syndrome (DIOS) within 6 months prior to enrollment.
- •Solid organ transplant or surgery affecting the large bowel other than appendectomy.
- •Small bowel surgery that significantly affected absorptive capacity (e.g. gastrectomy or pancreatectomy).
- •Intestinal inflammatory diseases including chronic diarrheal illness unrelated to pancreatic insufficiency.
- •Celiac disease or Crohn's disease.
- •Receiving enteral tube feeds for ≥50% of daily calorie intake.
- •Pregnancy or lactation.
- •Any type of malignancy involving the digestive tract in the last 5 years.
- •Known allergy to pancreatin or inactive ingredients (excipients) of pancreatin capsules.
- •Suspected non-compliance or non-cooperation.
- •Intake of experimental drugs within 30 days prior to study start.
- •Mental disability or any other lack of fitness, in the Investigator's opinion, to preclude subject's participation in or ability to complete the study.
- •Diagnosis of human immunodeficiency virus in medical history.
- •Listed for lung transplantation or other solid organ transplant or documented forced expiratory volume (FEV) ≤25%.
- •Use of lipid lowering therapy including statins, fibrates, niacin, and proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors that cannot be held at least 14 days prior to Day 1 and through Day 15 of the study.
结局指标
主要结局
Maximum serum triglyceride concentration
时间窗: 6 hours
Maximum serum triglyceride concentration during the 6 hour period following administration of the oral nutritional supplement.
Area under the curve (AUC) for triglyceride serum levels
时间窗: 6 hours
Incremental area under the curve (AUC) for triglyceride serum levels during the 6 hour time period following following administration of the oral nutritional supplement.
Change in serum triglyceride concentration
时间窗: 6 hours
Maximum change from baseline for serum triglyceride concentration based on an hourly blood draw over a 6 hour period following administration of the oral nutritional supplement.
次要结局
- Total cholesterol(6 hours)
- Serum glucose(6 hours)
- Incidence of gastrointestinal (GI) symptoms(6 hours)
- Number of stools and other abdominal symptoms(6 days)
- HDL cholesterol(6 hours)
- VLDL cholesterol(6 hours)
- LDL cholesterol(6 hours)