Evaluation of a 7-Day Therapeutic Trial Dose of Commercial Sucraid® (Sacrosidase) Oral Solution for Alleviating Congenital Sucrase-Isomaltase Deficiency (CSID) Symptoms in Pediatric Subjects With Low, Moderate, and Normal Sucrase Levels
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 入组人数
- 312
- 试验地点
- 20
- 主要终点
- Effects of Sucraid® treatment on gastrointestinal symptoms in subjects with low, moderate, and normal disaccharidase assay sucrase levels from an esophagogastroduodenoscopy (EGD).
研究概览
简要总结
This is a Phase 4, U.S. only, multi-center study using a 7-day therapeutic response dose (TRD) of commercial Sucraid® to assess the response of treatment in 1100 symptomatic pediatric (6 months to 17 years old) subjects with low, moderate, and normal sucrase activity determined by a disaccharidase assay via EGD within 1 year of the Screening Visit. This study will also explore the relationship between known genetic CSID mutations and sucrase activities via (EGD) disaccharidase assay (low, moderate, and normal).
详细描述
This phase 4 study will evaluate the response to Sucraid® in pediatric subjects aged 6 months to 17 years old with low (< 25 µM/min/gram protein), moderate (25-35 µM/min/gram protein), and normal (> 55 µM/min/gram protein) sucrase activities. Subjects with a sucrase level via disaccharidase assay from an EGD within 1 year of informed consent/assent, a normal histological interpretation, and at the discretion of the investigator, at least one symptom of carbohydrate maldigestion (CMS) of postprandial diarrhea, abdominal pain, gas/bloating, or defecation urgency at least 3 times per week for the past 3 months or more will be eligible for study participation. This study will also explore the relationship between three groups of sucrase levels from the EGD disaccharidase assay and the genetic test. This study will consist of a Screening Visit, Run-in Period, Baseline Visit, Treatment Period, and Follow-up Visit.
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
- 6 Months 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects with documented disaccharidase assay levels of lactase, sucrase, maltase, and palatinase via EGD disaccharidase assay (DA) performed no more than 1 year prior to enrollment with normal histological interpretation. Eligible subjects will be enrolled into the following study groups based on their documented sucrase level.
- •DA Sucrase < 25 (n=500)
- •DA Sucrase 25-35 (n=500)
- •DA Sucrase >55 (n=100)
- •At the discretion of the investigator, subject is suspected of CSID and has at least one symptom of diarrhea, abdominal pain, gas/bloating, nausea, or borborygmi at least 3x per week for the past 3 months or more.
- •Subject or parent/guardian must provide informed consent/assent prior to any study procedures being performed.
- •Subject is a U.S. resident.
- •Subject is male or female, ages 6 months to 17 years old.
- •Subject/parent or guardian is willing and able to complete necessary study procedures including following study instructions, completing electronic questionnaires via a personal mobile device, attending study visits and, in the Investigator's judgment, is sufficiently stable to participate in the study.
- •Subject/ parent or guardian must be able to read or understand the English language.
- •Subject or parent/guardian must have their own Android or Apple device for BYOD.
排除标准
- •Females who are lactating or pregnant.
- •Subjects with allergy to sucrose, yeast, papain, or glycerol.
- •Subjects with causes of abdominal pain or altered bowel habits other than CSID such as inflammatory bowel disease, celiac disease, eosinophilic gastrointestinal disorder, pancreatitis, or gastrointestinal bleeding.
- •Subjects with a history of diabetes mellitus.
- •Subjects with a recent febrile illness (5 days prior to study).
- •Subjects that do not have the mental capacity to understand the study requirements and are unable to comply.
- •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 previously used Sucraid®.
- •Subject has uncontrolled systematic disease.
研究组 & 干预措施
Single Arm Open Labeled Commercial Sucraid
All subjects will complete a 7-day treatment period of open-labeled FDA approved commercial Sucraid.
干预措施: Sucraid (sacrosidase) Oral Solution 8500 IU/mL (Biological)
结局指标
主要结局
Effects of Sucraid® treatment on gastrointestinal symptoms in subjects with low, moderate, and normal disaccharidase assay sucrase levels from an esophagogastroduodenoscopy (EGD).
时间窗: Post 7-day run-in period plus 7-day treatment period.
The primary endpoint will be the difference observed between the average symptom severity/frequency score during the 7-day treatment period and the 7-day run-in period as determined by the Daily Symptom Questionnaire using a scale from 0 to 5 or more.
次要结局
- Explore the relationship between known genetic CSID mutations and sucrase activities via (EGD) disaccharidase assay (low, moderate, and normal).(Up to 2 years)