Adult Pilot Study to Establish the Reference Ranges and Optimal Cut-offs for Oral 50g Sucrose Challenge Test & 13C-Sucrose Breath Test for CSID Cases (Previously Diagnosed Sucraid Responders) & Controls (GI Asymptomatic Healthy Volunteers)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 211
- 试验地点
- 1
- 主要终点
- Establish the normal reference range for the 50g Sucrose Challenge Test (SCT)
研究概览
简要总结
An adult pilot study to establish the normal reference range and optimal cutoffs for the oral 50g Sucrose Challenge Test (SCT) and the 50g 13C-Sucrose Breath Test (CBT) for patients with CSID and healthy controls. This study will enroll 120 healthy controls and 50 CSID patients. No study drug will be administered during this study.
详细描述
In an effort to mimic the real-world diagnostic setting for these tests, this study will be conducted virtually using a central site with telemedicine capabilities and electronic data capture software to collect data in real time via a mobile app. Subject will complete 3 visits within 21 days. Eligible subjects will undergo the following tests:
- 50g 13C-Sucrose Breath Test (CBT)
- Sucrose Intolerance Hydrogen Methane Breath Test (HBT)
- Sucrose Challenge Test (SCT)
- Buccal swab for CSID Genetic Testing (GT)
The primary aim is to establish the reference/normal ranges and optimal cutoff values for the SCT and CBT.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Subject must provide informed consent prior to any study procedures being performed.
- •Subject is a U.S. resident, and speaks and understands English
- •Subject is male or female, ≥ 18 years old.
- •CSID Cases must have been on Sucraid for at the last 12 months.
- •Healthy Volunteers do not have a CSID diagnosis or other chronic gastrointestinal conditions.
- •Subject must have their own Android or Apple device to access the mobile patient app.
排除标准
- •Females who are lactating or pregnant.
- •Subjects with allergy to sucrose.
- •Subjects with causes of abdominal pain or altered bowel habits other than symptoms -related to CSID (in CSID group) such as SIBO, IBD, celiac disease, pancreatitis, or -gastrointestinal bleeding.
- •Diabetes mellitus.
- •Use of systemic antibiotics, had a barium study, colonoscopy, unexplained runny diarrhea, or similar within 14 days prior to informed consent. Potential subjects can be re-screened once they meet this criterion.
- •Recent febrile illness.
- •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.
结局指标
主要结局
Establish the normal reference range for the 50g Sucrose Challenge Test (SCT)
时间窗: 4 hours post ingestion of sucrose
Reference ranges will be estimated by the lower 2.5th percentile and upper 97.5th percentile of the distribution of test results for the case and control populations.
Establish the optimal cutoff values for the 50g Sucrose Challenge Test (SCT)
时间窗: 4 hours post ingestion of sucrose
The mean and peak symptom severity score following sucrose ingestion will serve as markers of sucrase activity. The cutoff for the SCT will be determined by the maximum Youden Index that differentiates cases and controls.
Establish the normal reference range for the 50g 13C-Sucrose Breath Test (CBT)
时间窗: 4 hours post ingestion of sucrose
Reference ranges will be estimated by the lower 2.5th percentile and upper 97.5th percentile of the distribution of test results for the case and control populations.
Establish the optimal cutoff values for the 50g 13C-Sucrose Breath Test (CBT)
时间窗: 4 hours post ingestion of sucrose
The mean and peak symptom severity score following sucrose ingestion will serve as markers of sucrase activity. The cutoff for the SCT will be determined by the maximum Youden Index that differentiates cases and controls.
次要结局
- Compare the negative predictive value (NPV) of the 13C-Sucrose Breath Test (CBT) with the NPV of the Sucrose Hydrogen Methane Breath Test (HBT)(4 hours post ingestion of sucrose)
- Compare the positive predictive values (PPV) of the 13C-Sucrose Breath Test (CBT) with the positive predictive value of the Sucrose Hydrogen Methane Breath Test (HBT)(4 hours post ingestion of sucrose)
- Compare the negative predictive values (NPV) of the Sucrose Challenge Test (SCT) with the NPV of the Sucrose Hydrogen Methane Breath Test (HBT)(4 hours post ingestion of sucrose)
- Compare the positive predictive values (PPV) of the Sucrose Challenge Test (SCT) with the PPV of the Sucrose Hydrogen Methane Breath Test (HBT)(4 hours post ingestion of sucrose)