Clinical trial NCT05045495: Adult Pilot Study for Reference Ranges and Optimal Cut-offs for the Sucrose Challenge Test and Sucrose Breath Test
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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Congenital Sucrase-Isomaltase Deficiency
Keywords: CSID
Lead sponsor: QOL Medical, LLC
Sponsor class: INDUSTRY
First posted: 2021-09-16
Enrollment: 211
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Healthy volunteers and subjects with CSID.

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.

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:

1. 50g 13C-Sucrose Breath Test (CBT)
2. Sucrose Intolerance Hydrogen Methane Breath Test (HBT)
3. Sucrose Challenge Test (SCT)
4. 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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Establish the normal reference range for the 50g Sucrose Challenge Test (SCT)
timeFrame: 4 hours post ingestion of sucrose
measure: Establish the optimal cutoff values for the 50g Sucrose Challenge Test (SCT)
timeFrame: 4 hours post ingestion of sucrose
measure: Establish the normal reference range for the 50g 13C-Sucrose Breath Test (CBT)
timeFrame: 4 hours post ingestion of sucrose
measure: Establish the optimal cutoff values for the 50g 13C-Sucrose Breath Test (CBT)
timeFrame: 4 hours post ingestion of sucrose
Secondary outcomes:
measure: Compare the positive predictive values (PPV) of the Sucrose Challenge Test (SCT) with the PPV of the Sucrose Hydrogen Methane Breath Test (HBT)
timeFrame: 4 hours post ingestion of sucrose
measure: Compare the negative predictive values (NPV) of the Sucrose Challenge Test (SCT) with the NPV of the Sucrose Hydrogen Methane Breath Test (HBT)
timeFrame: 4 hours post ingestion of sucrose
measure: 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)
timeFrame: 4 hours post ingestion of sucrose
measure: Compare the negative predictive value (NPV) of the 13C-Sucrose Breath Test (CBT) with the NPV of the Sucrose Hydrogen Methane Breath Test (HBT)
timeFrame: 4 hours post ingestion of sucrose
Other outcomes:
Locations:
city: Culver City
country: United States
facility: Science 37-Recruiting Nationally
state: California
Study officials:
affiliation: QOL Medical
name: Weng Tao, M.D., Ph. D
role: PRINCIPAL_INVESTIGATOR
References: