An observational study in Congenital Sucrase-Isomaltase Deficiency, sponsored by QOL Medical, LLC. Completed at 1 site in United States. Open to participants aged 18 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2022-05-24.
Sponsored by QOL Medical, LLC · Observational
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.
QOL Medical, LLC is the lead sponsor of 5 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Healthy volunteers and subjects with CSID.
Exclusion Criteria:
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.
Diagnostic Test: Sucrose Hydrogen Methane Breath Test
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.
Diagnostic Test: Sucrose Hydrogen Methane Breath 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.
Also known as: 13C-Sucrose Breath Test, Sucrase Genetic Test (Buccal Swab), Sucrose Challenge Test
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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)
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.
Time frame: 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)
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.
Time frame: 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.
Time frame: 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.
Time frame: 4 hours post ingestion of sucrose
This study is completed, as verified in May 2022. You cannot join it, but the record below documents what was studied.
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