7-Day Trial of Sucraid for Alleviating CSID Symptoms in Subjects With Low, Moderate, and Normal Sucrase Levels

NCT05480761 | Terminated Phase 4 FDA Drug

• 1 other identifier: SSDXP-13
• Study Type: interventional
• Target: 312
• Locations: 1 country
• Sites: 20

Brief Summary

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).

Conditions

Congenital Sucrase-Isomaltase Deficiency; CSID; Sucrase Isomaltase Deficiency

Keywords

www.tummyachestudy.com

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Explore the relationship between known genetic CSID mutations and sucrase activities via (EGD) disaccharidase assay (low, moderate, and normal).

  Up to 2 years

Study Arms (1)

Single Arm Open Labeled Commercial Sucraid

EXPERIMENTAL

All subjects will complete a 7-day treatment period of open-labeled FDA approved commercial Sucraid.

Biological: Sucraid (sacrosidase) Oral Solution 8500 IU/mL

Interventions

Sucraid (sacrosidase) Oral Solution 8500 IU/mL BIOLOGICAL

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.

Also known as: Sucraid, Sacrosidase

Single Arm Open Labeled Commercial Sucraid

Eligibility Criteria

• Age: 6 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• QOL Medical, LLC: lead

Study Sites (20)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

Kidz Medical

Coral Gables, Florida, 33146, United States

Location

Orlando Health

Orlando, Florida, 32806, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30329, United States

Location

Center for Digestive Health Care, LLC

Atlanta, Georgia, 30342, United States

Location

University of Maryland Baltimore

Baltimore, Maryland, 21201, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

GI Associates

Flowood, Mississippi, 39232, United States

Location

Happy Tummies

Flowood, Mississippi, 39232, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Atlantic Health System

Morristown, New Jersey, 07960, United States

Location

WNY Pediatric Gastroenterology

Buffalo, New York, 14225, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

Measurable Outcomes Research

Oklahoma City, Oklahoma, 73112, United States

Location

Prisma Health

Greenville, South Carolina, 29615, United States

Location

GI For Kids, PLLC

Knoxville, Tennessee, 32963, United States

Location

Newco 3A Research,LLC DBA 3A Research

El Paso, Texas, 79902, United States

Location

Pediatric GI of El Paso, LLC

El Paso, Texas, 79925, United States

Location

McGovern Medical School of UT Health

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Sucrase-isomaltase deficiency, congenital

Interventions

beta-Fructofuranosidase; Solutions

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Pharmaceutical Preparations

Study Officials

• Weng Tao, M.D., Ph. D

  QOL Medical

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 28, 2022
• First Posted: July 29, 2022
• Study Start: August 1, 2022
• Primary Completion: September 18, 2024
• Study Completion: September 18, 2024
• Last Updated: March 20, 2025

Record last verified: 2023-03

Locations

US (20)