Congenital Sucrase-Isomaltase DeficiencyNCT05045495
Adult Pilot Study for Reference Ranges and Optimal Cut-offs for the Sucrose Challenge Test and Sucrose Breath Test
An observational study of Congenital Sucrase-Isomaltase Deficiency, run by QOL Medical, LLC at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Congenital Sucrase-Isomaltase Deficiency
- What is being tested?
- Sucrose Hydrogen Methane Breath Test
- How many people
- 211 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Aug 2021to 28 Feb 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Healthy volunteers and subjects with CSID.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Science 37-Recruiting Nationally
Culver City, California, 90230
About this study
Official title: 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)
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.
Read the detailed description
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.
- Conditions
- Congenital Sucrase-Isomaltase Deficiency
- Being tested
- Sucrose Hydrogen Methane Breath Test
- Keywords
- CSID
- Medical subject headings
- Sucrase-isomaltase deficiency, congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sucrose Hydrogen Methane Breath TestDiagnostic test
Also called 13C-Sucrose Breath Test, Sucrase Genetic Test (Buccal Swab), Sucrose Challenge 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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Diagnostic Test: Sucrose Hydrogen Methane Breath Test | |
| 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. Receives: Diagnostic Test: Sucrose Hydrogen Methane Breath Test |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 4 hours post ingestion of sucrose
Secondary outcomes (4)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 4 hours post ingestion of sucrose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2021
- Study start30 Aug 2021
- Primary completion28 Feb 2022
- Study completion28 Feb 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Sucrase-Isomaltase DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 211 participants (actual)
Sponsor and investigators
- Lead sponsor
- QOL Medical, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Weng Tao, M.D., Ph. D, Principal investigator, QOL Medical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05045495
- Sponsor's protocol ID
- SSDXA-12
- Status last verified
- May 2022
- First submitted
- 7 Sep 2021
- Last updated
- 24 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT05045495
- Acronym / sponsor ID
- SSDXA-12
- Conditions
- Congenital Sucrase-Isomaltase Deficiency
- Keywords
- CSID
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 211 participants (actual)
- Lead sponsor
- QOL Medical, LLCIndustry (pharma, biotech, devices)
- Study start
- 30 Aug 2021 (actual)
- Primary completion
- 28 Feb 2022 (actual)
- Study completion
- 28 Feb 2022 (actual)
- First posted
- 16 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Healthy Volunteer | – | Diagnostic Test: Sucrose Hydrogen Methane Breath Test120 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. |
| CSID Case | – | Diagnostic Test: Sucrose Hydrogen Methane Breath Test50 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Healthy volunteers and subjects with CSID.
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sucrose Hydrogen Methane Breath TestAlso: 13C-Sucrose Breath Test, Sucrase Genetic Test (Buccal Swab), Sucrose Challenge TestSubjects 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. | Diagnostic test | CSID Case, Healthy Volunteer |
Primary outcome
Establish the normal reference range for the 50g Sucrose Challenge Test (SCT)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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.
4 secondary outcomes
Compare the positive predictive values (PPV) of the Sucrose Challenge Test (SCT) with the PPV of the Sucrose Hydrogen Methane Breath Test (HBT)
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)
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)
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)
Time frame: 4 hours post ingestion of sucrose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 May 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Weng Tao, M.D., Ph. D principal investigator
QOL Medical
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Sucrase-Isomaltase Deficiency, run by QOL Medical, LLC at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Establish the normal reference range for the 50g Sucrose Challenge Test (SCT) (time frame: 4 hours post ingestion of sucrose). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 May 2022; AzTrials last refreshed its copy on 30 Oct 2026.