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).
This phase 4 study will evaluate the response to Sucraid® in pediatric subjects aged 6 months to 17 years old with low (\< 25 µM/min/gram protein), moderate (25-35 µM/min/gram protein), and normal (\> 55 µM/min/gram protein) sucrase activities. Subjects with a sucrase level via disaccharidase assay from an EGD within 1 year of informed consent/assent, a normal histological interpretation, and at the discretion of the investigator, at least one symptom of carbohydrate maldigestion (CMS) of postprandial diarrhea, abdominal pain, gas/bloating, or defecation urgency at least 3 times per week for the past 3 months or more will be eligible for study participation. This study will also explore the relationship between three groups of sucrase levels from the EGD disaccharidase assay and the genetic test. This study will consist of a Screening Visit, Run-in Period, Baseline Visit, Treatment Period, and Follow-up Visit.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
312
Sucraid is a pale yellow to colorless, clear solution of glycerol, water, and citric acid, with a pleasant, sweet taste. Each milliliter of Sucraid contains 8,500 IU of the enzyme sacrosidase, the active ingredient.
Children's Hospital Los Angeles
Los Angeles, California, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States
Kidz Medical
Coral Gables, Florida, United States
Orlando Health
Orlando, Florida, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Center for Digestive Health Care, LLC
Atlanta, Georgia, United States
University of Maryland Baltimore
Baltimore, Maryland, United States
Johns Hopkins University
Baltimore, Maryland, United States
GI Associates
Flowood, Mississippi, United States
Happy Tummies
Flowood, Mississippi, United States
...and 10 more locations
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.
Time frame: Post 7-day run-in period plus 7-day treatment period.
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.
Time frame: Up to 2 years
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