A Multicenter, Double-Blind, Placebo-Controlled Trial to Evaluate the Frequency of Genetic Sucrase-Isomaltase Deficiency Genotypes, and the Efficacy and Safety of Sucraid® (Sacrosidase) Oral Solution in Subjects With Chronic Diarrhea and Sucrase Deficiency
试验速览
- 阶段
- 4 期
- 状态
- 撤回
- 主要终点
- Response to Sucraid and placebo in a parallel group study based on improvement in daily stool consistency, as assessed by the BSFS using SHMBT
研究概览
简要总结
S09A is a Phase 4, multicenter, randomized, double-blind, placebo-controlled, parallel study examining the efficacy and safety of a Sucraid (sacrosidase) Oral Solution in comparison to a placebo in 150-200 subjects with chronic diarrhea possibly attributable to sucrase deficiency.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject is 16 years of age or older.
- •Subject is male or female. Women of childbearing potential must be willing to use one of the following contraception methods (for at least 10 days prior to start of study drug and for 10 to 14 days after last dose of study drug): Oral contraceptive, Injectable progestogen, Implants of levonorgestrel, Estrogenic vaginal ring, Percutaneous contraceptive patches, Intrauterine device, Sterile male partner, Double-barrier method of contraception Women of non-child bearing potential include females regardless of age with functioning ovaries and who have a current tubal ligation (Hatcher, 2004), bilateral oophorectomy, or total hysterectomy, or post-menopausal females. Note: Post-menopausal is defined as 1 year without menses with an appropriate clinical profile (e.g., age appropriate, >45 years, in the absence of hormone replacement therapy).
- •Subject has a minimum of 3 months of self-reported diarrhea (BSFS scores ≥ 5 on at least 3 days per week and ≥1 stool per day)
- •Subject has a value in the SHMBT of at least 20 ppm for hydrogen, or 12 ppm for methane or 15 ppm above a previous breath sample for the combination of both gases.
- •Subject reports that he/she experienced soft stools or diarrhea within the last 24 hours when contacted by the site 24 hours after completing the SHMBT.
- •Subject is able to read, speak, and verbally understand the English language.
- •Subject is located in the United States.
- •Subject has access to the Internet on a daily basis.
- •Subject has access to an acceptable Apple iPhone/iPad/iTouch or Android smartphone/tablet. The sponsor may choose to provide a smartphone in unusual cases (please contact sponsor to request loaner device when applicable)
排除标准
- •Subject has recent history of functional or chronic constipation.
- •Subject has known history of ulcerative colitis, Crohn's disease, or Celiac disease.
- •Subject has known hypersensitivity to papain, glycerol, or yeast.
- •Subject has received bovine serum in the last year.
- •Subject has previous history of Sucraid use.
- •Subject has taken any prebiotic or probiotic within 5 days prior to Visit 2 and does not agree to refrain from taking them during the study.
- •Subject is female and is pregnant, breastfeeding, or planning to become pregnant during the study.
- •Subject has known uncontrolled systemic disease.
- •Subject has prior diagnosis of Type 1 or Type 2 diabetes.
- •Subject has history of bowel resection.
- •Subject is undergoing chemotherapy for the treatment of cancer.
- •Subject has major physical or psychiatric illness within the last 6 months that in the opinion of the investigator would affect the subject's ability to complete the trial.
- •Subject has used an investigational device or investigational drug within 30 days prior to Visit 1.
研究组 & 干预措施
Treatment
Subjects randomized to the active treatment arm will take Sucraid, 2 mL (17,000 IU) with every meal or snack, administered orally following dilution with 2 to 4 ounces (60 to 120 mL) of water or milk (either cold or at room temperature). The study treatment period is one week.
干预措施: Sucraid (Drug)
Placebo
Subjects randomized to the placebo treatment arm will take Sucraid placebo, 2 mL (17,000 IU) with every meal or snack, administered orally following dilution with 2 to 4 ounces (60 to 120 mL) of water or milk (either cold or at room temperature). The study treatment period is one week.
干预措施: Placebo (Drug)
结局指标
主要结局
Response to Sucraid and placebo in a parallel group study based on improvement in daily stool consistency, as assessed by the BSFS using SHMBT
时间窗: Up to 2 years
Response to Sucraid and placebo based on improvement in daily stool consistency, as assessed by the Bristol Stool Form Scale (BSFS) over a 1-week treatment period in subjects with chronic diarrhea and sucrase deficiency using a sucrose hydrogen methane breath test (SHMBT).
次要结局
- Effects of Sucraid and placebo on daily stool frequency(Up to 2 years)
- The relationship between the severity of sucrase deficiency, quantified by a SHMBT(Up to 2 years)
- The allele frequency of the most common sucrase-isomaltase genetic variants in subjects with chronic diarrhea attributable to sucrase deficiency compared to the allele frequency in other databases(Up to 2 years)
- Effects of Sucraid and placebo on daily assessments of Bristol Stool Form Scale(Up to 2 years)
- Effects of Sucraid and placebo on daily abdominal pain(Up to 2 years)
- Effects of Sucraid and placebo on daily bloating severity(Up to 2 years)
- The mean improvement in the BSFS for each treatment group.(Up to 2 years)
- Overall frequency of the 4 most common sucrase-isomaltase deficiency genetic variants(Up to 2 years)
- The number of less common sucrase-isomaltase polymorphisms in this study population.(Up to 2 years)