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

November 14, 2023 updated by: QOL Medical, LLC

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

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

1100

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital Los Angeles
        • Contact:
          • Ani Antaramian
        • Principal Investigator:
          • Rohit Kohli, MD
      • Oakland, California, United States, 94609
        • Recruiting
        • UCSF Benioff Children's Hospital Oakland
        • Contact:
          • Emily Stekol
          • Phone Number: 510-428-3058
        • Contact:
          • Courtney Carter
          • Phone Number: 510-428-3058
        • Principal Investigator:
          • Namrata Patel-Sanchez, MD
    • Florida
      • Coral Gables, Florida, United States, 33146
        • Recruiting
        • Kidz Medical
        • Contact:
          • Minor Gerard
        • Principal Investigator:
          • Tano Mario, MD
      • Orlando, Florida, United States, 32806
        • Recruiting
        • Orlando Health
        • Contact:
          • Katelyn Kern
        • Contact:
          • Linda Tilme
        • Principal Investigator:
          • Devendra Mehta, MD
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Recruiting
        • Children's Healthcare of Atlanta
        • Contact:
          • Angela Stallworth
          • Phone Number: 404-785-9400
        • Principal Investigator:
          • Hillary Bashaw, MD
      • Atlanta, Georgia, United States, 30342
        • Recruiting
        • Center for Digestive Health Care, LLC
        • Contact:
          • Nitin Rastogi
        • Principal Investigator:
          • Stanley A Cohen, MD
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Recruiting
        • Johns Hopkins University
        • Contact:
          • Ashley Thomas, RN
          • Phone Number: 410-955-8769
        • Principal Investigator:
          • Shaija Kutty, MD
      • Baltimore, Maryland, United States, 21201
        • Recruiting
        • University of Maryland Baltimore
        • Contact:
          • Valerie Weir
          • Phone Number: 410-706-3100
        • Principal Investigator:
          • Runa Watkins, MD
    • Mississippi
      • Flowood, Mississippi, United States, 39232
        • Recruiting
        • Happy Tummies
        • Contact:
          • Calandra Brown
        • Principal Investigator:
          • Angela Shannon, MD
      • Flowood, Mississippi, United States, 39232
        • Recruiting
        • GI Associates
        • Contact:
        • Principal Investigator:
          • Mary A Lowry, MD
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Recruiting
        • Children's Mercy Hospital
        • Contact:
          • Erin Payne
          • Phone Number: 816-234-3066
        • Principal Investigator:
          • Craig Friesen, MD
    • New Jersey
      • Morristown, New Jersey, United States, 07960
    • New York
      • Buffalo, New York, United States, 14225
        • Recruiting
        • WNY Pediatric Gastroenterology
        • Contact:
          • Hashmi Yusuf
        • Principal Investigator:
          • Daniel Gelfond, MD
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • University Hospitals Cleveland Medical Center
        • Contact:
          • Kelsey Lesniak, RN
          • Phone Number: 216-844-1765
        • Principal Investigator:
          • Thomas Sferra, MD
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73112
        • Recruiting
        • Measurable Outcomes Research
        • Contact:
          • Margaret Freede
        • Principal Investigator:
          • Maryam Shambayati, MD
    • South Carolina
      • Greenville, South Carolina, United States, 29615
        • Recruiting
        • Prisma Health
        • Contact:
          • Carrie Frost
        • Principal Investigator:
          • Jonathan Markowitz, M.D.
    • Tennessee
      • Knoxville, Tennessee, United States, 32963
        • Recruiting
        • GI For Kids, PLLC
        • Principal Investigator:
          • Youhanna Al-Tawil, MD
        • Contact:
    • Texas
      • El Paso, Texas, United States, 79902
        • Recruiting
        • Newco 3A Research,LLC DBA 3A Research
        • Contact:
        • Principal Investigator:
          • Aldo Maspons, MD
      • El Paso, Texas, United States, 79925
        • Recruiting
        • Pediatric GI of El Paso, LLC
        • Contact:
        • Principal Investigator:
          • Eduardo D. Rosas-Blum, MD
      • Houston, Texas, United States, 77030
        • Recruiting
        • McGovern Medical School of UT Health
        • Contact:
        • Principal Investigator:
          • Melissa Van Arsdall, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 17 years (Child)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Single Arm Open Labeled Commercial Sucraid
All subjects will complete a 7-day treatment period of open-labeled FDA approved commercial Sucraid.
Biological: 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.
Other Names:
  • Sacrosidase
  • Sucraid

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Effects of Sucraid® treatment on gastrointestinal symptoms in subjects with low, moderate, and normal disaccharidase assay sucrase levels from an esophagogastroduodenoscopy (EGD).
Time Frame: Post 7-day run-in period plus 7-day treatment period.
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Explore the relationship between known genetic CSID mutations and sucrase activities via (EGD) disaccharidase assay (low, moderate, and normal).
Time Frame: Up to 2 years
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

QOL Medical, LLC

Investigators

  • Principal Investigator: Weng Tao, M.D., Ph. D, QOL Medical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 1, 2022

Primary Completion (Estimated)

August 25, 2024

Study Completion (Estimated)

December 25, 2024

Study Registration Dates

First Submitted

June 28, 2022

First Submitted That Met QC Criteria

July 27, 2022

First Posted (Actual)

July 29, 2022

Study Record Updates

Last Update Posted (Actual)

November 15, 2023

Last Update Submitted That Met QC Criteria

November 14, 2023

Last Verified

March 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SSDXP-13

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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