Sucrase Isomaltase Deficiency Clinical Trial
Official title:
Evaluation of a 7-Day Therapeutic Trial Dose of Commercial Sucraid® (Sacrosidase) Oral Solution for Alleviating Congenital Sucrase-Isomaltase Deficiency (CSID) Symptoms in Pediatric Subjects With Low, Moderate, and Normal Sucrase Levels
| Verified date | March 2023 |
| Source | QOL Medical, LLC |
| Contact | Kristy Wort |
| Phone | 972-273-9012 |
| kwort[@]totalcro.com | |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
This is a Phase 4, U.S. only, multi-center study using a 7-day therapeutic response dose (TRD) of commercial Sucraid® to assess the response of treatment in 1100 symptomatic pediatric (6 months to 17 years old) subjects with low, moderate, and normal sucrase activity determined by a disaccharidase assay via EGD within 1 year of the Screening Visit. This study will also explore the relationship between known genetic CSID mutations and sucrase activities via (EGD) disaccharidase assay (low, moderate, and normal).
| Status | Recruiting |
| Enrollment | 1100 |
| Est. completion date | December 25, 2024 |
| Est. primary completion date | August 25, 2024 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 6 Months to 17 Years |
| Eligibility | Inclusion Criteria: - Subjects with documented disaccharidase assay levels of lactase, sucrase, maltase, and palatinase via EGD disaccharidase assay (DA) performed no more than 1 year prior to enrollment with normal histological interpretation. Eligible subjects will be enrolled into the following study groups based on their documented sucrase level. - DA Sucrase < 25 (n=500) - DA Sucrase 25-35 (n=500) - DA Sucrase >55 (n=100) - At the discretion of the investigator, subject is suspected of CSID and has at least one symptom of diarrhea, abdominal pain, gas/bloating, nausea, or borborygmi at least 3x per week for the past 3 months or more. - Subject or parent/guardian must provide informed consent/assent prior to any study procedures being performed. - Subject is a U.S. resident. - Subject is male or female, ages 6 months to 17 years old. - Subject/parent or guardian is willing and able to complete necessary study procedures including following study instructions, completing electronic questionnaires via a personal mobile device, attending study visits and, in the Investigator's judgment, is sufficiently stable to participate in the study. - Subject/ parent or guardian must be able to read or understand the English language. - Subject or parent/guardian must have their own Android or Apple device for BYOD. Exclusion Criteria: - Females who are lactating or pregnant. - Subjects with allergy to sucrose, yeast, papain, or glycerol. - Subjects with causes of abdominal pain or altered bowel habits other than CSID such as inflammatory bowel disease, celiac disease, eosinophilic gastrointestinal disorder, pancreatitis, or gastrointestinal bleeding. - Subjects with a history of diabetes mellitus. - Subjects with a recent febrile illness (5 days prior to study). - Subjects that do not have the mental capacity to understand the study requirements and are unable to comply. - 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. - Subject has previously used Sucraid®. - Subject has uncontrolled systematic disease. |
| Country | Name | City | State |
|---|---|---|---|
| United States | Center for Digestive Health Care, LLC | Atlanta | Georgia |
| United States | Children's Healthcare of Atlanta | Atlanta | Georgia |
| United States | Johns Hopkins University | Baltimore | Maryland |
| United States | University of Maryland Baltimore | Baltimore | Maryland |
| United States | WNY Pediatric Gastroenterology | Buffalo | New York |
| United States | University Hospitals Cleveland Medical Center | Cleveland | Ohio |
| United States | Kidz Medical | Coral Gables | Florida |
| United States | Newco 3A Research,LLC DBA 3A Research | El Paso | Texas |
| United States | Pediatric GI of El Paso, LLC | El Paso | Texas |
| United States | GI Associates | Flowood | Mississippi |
| United States | Happy Tummies | Flowood | Mississippi |
| United States | Prisma Health | Greenville | South Carolina |
| United States | McGovern Medical School of UT Health | Houston | Texas |
| United States | Children's Mercy Hospital | Kansas City | Missouri |
| United States | GI For Kids, PLLC | Knoxville | Tennessee |
| United States | Children's Hospital Los Angeles | Los Angeles | California |
| United States | Atlantic Health System | Morristown | New Jersey |
| United States | UCSF Benioff Children's Hospital Oakland | Oakland | California |
| United States | Measurable Outcomes Research | Oklahoma City | Oklahoma |
| United States | Orlando Health | Orlando | Florida |
| Lead Sponsor | Collaborator |
|---|---|
| QOL Medical, LLC |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Effects of Sucraid® treatment on gastrointestinal symptoms in subjects with low, moderate, and normal disaccharidase assay sucrase levels from an esophagogastroduodenoscopy (EGD). | The primary endpoint will be the difference observed between the average symptom severity/frequency score during the 7-day treatment period and the 7-day run-in period as determined by the Daily Symptom Questionnaire using a scale from 0 to 5 or more. | Post 7-day run-in period plus 7-day treatment period. | |
| Secondary | Explore the relationship between known genetic CSID mutations and sucrase activities via (EGD) disaccharidase assay (low, moderate, and normal). | The number and percentage of subjects with genetic CSID mutations will also be examined in low, moderate, and normal sucrase study group. | Up to 2 years |