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

NCT ID: NCT05480761

Last Updated: 2025-03-20

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 312 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-08-01
• Study Completion Date: 2024-09-18

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).

Detailed Description

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.

Conditions

Congenital Sucrase-Isomaltase Deficiency; CSID; Sucrase Isomaltase Deficiency

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Single Arm Open Labeled Commercial Sucraid

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

Group Type: EXPERIMENTAL

Sucraid (sacrosidase) Oral Solution 8500 IU/mL

Intervention Type: 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.

Interventions

Sucraid (sacrosidase) Oral Solution 8500 IU/mL

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.

Intervention Type: BIOLOGICAL

Other Intervention Names

Sucraid; Sacrosidase

Eligibility Criteria

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.

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

QOL Medical, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Weng Tao, M.D., Ph. D

Role: PRINCIPAL_INVESTIGATOR

QOL Medical

Locations

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

Children's Mercy Hospital

Kansas City, Missouri, United States

Atlantic Health System

Morristown, New Jersey, United States

WNY Pediatric Gastroenterology

Buffalo, New York, United States

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Measurable Outcomes Research

Oklahoma City, Oklahoma, United States

Prisma Health

Greenville, South Carolina, United States

GI For Kids, PLLC

Knoxville, Tennessee, 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

McGovern Medical School of UT Health

Houston, Texas, United States

Countries

United States

Other Identifiers

SSDXP-13

Identifier Type: -

Identifier Source: org_study_id