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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.
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:
The primary aim is to establish the reference/normal ranges and optimal cutoff values for the SCT and CBT.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Healthy Volunteer | 120 Healthy Volunteers will perform the 50-gram sucrose challenge test, Sucrose hydrogen methane breath test, 13C-sucrose breath test and collect buccal swab samples for genetic testing. |
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| CSID Case | 50 CSID cases defined by being on Sucraid for at least 12 months will perform the 50-gram sucrose challenge test, Sucrose hydrogen methane breath test, 13C-sucrose breath test and collect buccal swab samples for genetic testing. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sucrose Hydrogen Methane Breath Test | Diagnostic Test | Subjects will collect 2 buccal swabs for the sucrase genetic test and then subjects will simultaneously conduct the sucrose challenge test, sucrose hydrogen methane breath test, and the 13C-sucrose following ingestion of 50-grams of sucrose (table sugar) mixed with 8 ounce of water. |
| Measure | Description | Time Frame |
|---|---|---|
| Establish the normal reference range for the 50g Sucrose Challenge Test (SCT) | 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. | 4 hours post ingestion of sucrose |
| Establish the optimal cutoff values for the 50g Sucrose Challenge Test (SCT) | 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. | 4 hours post ingestion of sucrose |
| Establish the normal reference range for the 50g 13C-Sucrose Breath Test (CBT) | 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. | 4 hours post ingestion of sucrose |
| Establish the optimal cutoff values for the 50g 13C-Sucrose Breath Test (CBT) | 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. | 4 hours post ingestion of sucrose |
| Measure | Description | Time Frame |
|---|---|---|
| Compare the positive predictive values (PPV) of the Sucrose Challenge Test (SCT) with the PPV of the Sucrose Hydrogen Methane Breath Test (HBT) | PPV of SCT relative to the HBT and percentage of Sucraid® Responders in CSID cases and asymptomatic controls, and in cases confirmed by each of the following three diagnostic tools: HBT, CBT, and SCT. | 4 hours post ingestion of sucrose |
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Inclusion Criteria:
Exclusion Criteria:
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Healthy volunteers and subjects with CSID.
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| Name | Affiliation | Role |
|---|---|---|
| Weng Tao, M.D., Ph. D | QOL Medical | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Science 37-Recruiting Nationally | Culver City | California | 90230 | United States |
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| ID | Term |
|---|---|
| C538139 | Sucrase-isomaltase deficiency, congenital |
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Cheek cell samples via buccal swab
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| Compare the negative predictive values (NPV) of the Sucrose Challenge Test (SCT) with the NPV of the Sucrose Hydrogen Methane Breath Test (HBT) | NPV of SCT relative to the HBT and percentage of Sucraid® Responders in CSID cases and asymptomatic controls, and in cases confirmed by each of the following three diagnostic tools: HBT, CBT, and SCT. | 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) | PPV of the CBT relative to the HBT and percentage of Sucraid® Responders in CSID cases and asymptomatic controls, and in cases confirmed by each of the following three diagnostic tools: HBT, CBT, and SCT. | 4 hours post ingestion of sucrose |
| Compare the negative predictive value (NPV) of the 13C-Sucrose Breath Test (CBT) with the NPV of the Sucrose Hydrogen Methane Breath Test (HBT) | NPV of the CBT relative to the HBT and percentage of Sucraid® Responders in CSID cases and asymptomatic controls, and in cases confirmed by each of the following three diagnostic tools: HBT, CBT, and SCT. | 4 hours post ingestion of sucrose |