Adult Pilot Study for Reference Ranges and Optimal Cut-offs for the Sucrose Challenge Test and Sucrose Breath Test

NCT05045495 | Completed

• 1 other identifier: SSDXA-12
• Study Type: observational
• Target: 211
• Locations: 1 country
• Sites: 1

Brief 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. No study drug will be administered during this study.

Conditions

Congenital Sucrase-Isomaltase Deficiency

Keywords

CSID

Outcome Measures

Primary Outcomes (4)

• 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

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)

  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 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 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

Study Arms (2)

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.

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.

Diagnostic Test: Sucrose Hydrogen Methane Breath Test

Interventions

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.

Also known as: 13C-Sucrose Breath Test, Sucrase Genetic Test (Buccal Swab), Sucrose Challenge Test

CSID Case; Healthy Volunteer

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Healthy volunteers and subjects with CSID.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• QOL Medical, LLC: lead

Study Sites (1)

Science 37-Recruiting Nationally

Culver City, California, 90230, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Cheek cell samples via buccal swab

MeSH Terms

Conditions

Sucrase-isomaltase deficiency, congenital

Study Officials

• Weng Tao, M.D., Ph. D

  QOL Medical

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 7, 2021
• First Posted: September 16, 2021
• Study Start: August 30, 2021
• Primary Completion: February 28, 2022
• Study Completion: February 28, 2022
• Last Updated: May 24, 2022

Record last verified: 2022-05

Locations

US (1)