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

Primary Purpose

Congenital Sucrase-Isomaltase Deficiency, CSID, Sucrase Isomaltase Deficiency

Status
Recruiting
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Sucraid (sacrosidase) Oral Solution 8500 IU/mL
Sponsored by
QOL Medical, LLC
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Sucrase-Isomaltase Deficiency focused on measuring www.tummyachestudy.com

Eligibility Criteria

6 Months - 17 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital Los AngelesRecruiting
  • UCSF Benioff Children's Hospital OaklandRecruiting
  • Kidz MedicalRecruiting
  • Orlando HealthRecruiting
  • Children's Healthcare of AtlantaRecruiting
  • Center for Digestive Health Care, LLCRecruiting
  • University of Maryland BaltimoreRecruiting
  • Johns Hopkins UniversityRecruiting
  • GI AssociatesRecruiting
  • Happy TummiesRecruiting
  • Children's Mercy HospitalRecruiting
  • Atlantic Health SystemRecruiting
  • WNY Pediatric GastroenterologyRecruiting
  • University Hospitals Cleveland Medical CenterRecruiting
  • Measurable Outcomes ResearchRecruiting
  • Prisma HealthRecruiting
  • GI For Kids, PLLCRecruiting
  • Newco 3A Research,LLC DBA 3A ResearchRecruiting
  • Pediatric GI of El Paso, LLCRecruiting
  • McGovern Medical School of UT HealthRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Single Arm Open Labeled Commercial Sucraid

Arm Description

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

Full Information

First Posted
June 28, 2022
Last Updated
March 27, 2023
Sponsor
QOL Medical, LLC
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1. Study Identification

Unique Protocol Identification Number
NCT05480761
Brief Title
7-Day Trial of Sucraid for Alleviating CSID Symptoms in Subjects With Low, Moderate, and Normal Sucrase Levels
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 1, 2022 (Actual)
Primary Completion Date
August 25, 2024 (Anticipated)
Study Completion Date
December 25, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
QOL Medical, LLC

4. Oversight

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
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Sucrase-Isomaltase Deficiency, CSID, Sucrase Isomaltase Deficiency
Keywords
www.tummyachestudy.com

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1100 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Single Arm Open Labeled Commercial Sucraid
Arm Type
Experimental
Arm Description
All subjects will complete a 7-day treatment period of open-labeled FDA approved commercial Sucraid.
Intervention Type
Biological
Intervention Name(s)
Sucraid (sacrosidase) Oral Solution 8500 IU/mL
Other Intervention Name(s)
Sucraid, Sacrosidase
Intervention Description
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.
Primary Outcome Measure Information:
Title
Effects of Sucraid® treatment on gastrointestinal symptoms in subjects with low, moderate, and normal disaccharidase assay sucrase levels from an esophagogastroduodenoscopy (EGD).
Description
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.
Secondary Outcome Measure Information:
Title
Explore the relationship between known genetic CSID mutations and sucrase activities via (EGD) disaccharidase assay (low, moderate, and normal).
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Tanuja Saxena
Phone
214-855-1222
Email
tsaxena@totalcro.com
First Name & Middle Initial & Last Name or Official Title & Degree
Kasidy Street
Phone
919-832-8585
Email
kstreet@qolmed.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Weng Tao, M.D., Ph. D
Organizational Affiliation
QOL Medical
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ani Antaramian
First Name & Middle Initial & Last Name & Degree
Rohit Kohli, MD
Facility Name
UCSF Benioff Children's Hospital Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Emily Stekol
Phone
510-428-3058
First Name & Middle Initial & Last Name & Degree
Courtney Carter
Phone
510-428-3058
First Name & Middle Initial & Last Name & Degree
Namrata Patel-Sanchez, MD
Facility Name
Kidz Medical
City
Coral Gables
State/Province
Florida
ZIP/Postal Code
33146
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Minor Gerard
First Name & Middle Initial & Last Name & Degree
Tano Mario, MD
Facility Name
Orlando Health
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Katelyn Kern
First Name & Middle Initial & Last Name & Degree
Linda Tilme
First Name & Middle Initial & Last Name & Degree
Devendra Mehta, MD
Facility Name
Children's Healthcare of Atlanta
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Angela Stallworth
Phone
404-785-9400
First Name & Middle Initial & Last Name & Degree
Hillary Bashaw, MD
Facility Name
Center for Digestive Health Care, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30342
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nitin Rastogi
First Name & Middle Initial & Last Name & Degree
Stanley A Cohen, MD
Facility Name
University of Maryland Baltimore
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Valerie Weir
Phone
410-706-3100
First Name & Middle Initial & Last Name & Degree
Runa Watkins, MD
Facility Name
Johns Hopkins University
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ashley Thomas, RN
Phone
410-955-8769
First Name & Middle Initial & Last Name & Degree
Shaija Kutty, MD
Facility Name
GI Associates
City
Flowood
State/Province
Mississippi
ZIP/Postal Code
39232
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nolan Runnels
Phone
601-863-0395
Email
nolan.runnels@gastrodocs.net
First Name & Middle Initial & Last Name & Degree
Mary A Lowry, MD
Facility Name
Happy Tummies
City
Flowood
State/Province
Mississippi
ZIP/Postal Code
39232
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Calandra Brown
First Name & Middle Initial & Last Name & Degree
Angela Shannon, MD
Facility Name
Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Erin Payne
Phone
816-234-3066
First Name & Middle Initial & Last Name & Degree
Craig Friesen, MD
Facility Name
Atlantic Health System
City
Morristown
State/Province
New Jersey
ZIP/Postal Code
07960
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Annette Langseder, RN
Phone
973-971-5676
Email
annette.langseder@atlantichealth.org
First Name & Middle Initial & Last Name & Degree
Barbara Verga, MD
Facility Name
WNY Pediatric Gastroenterology
City
Buffalo
State/Province
New York
ZIP/Postal Code
14225
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hashmi Yusuf
First Name & Middle Initial & Last Name & Degree
Daniel Gelfond, MD
Facility Name
University Hospitals Cleveland Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kelsey Lesniak, RN
Phone
216-844-1765
First Name & Middle Initial & Last Name & Degree
Thomas Sferra, MD
Facility Name
Measurable Outcomes Research
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73112
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Margaret Freede
First Name & Middle Initial & Last Name & Degree
Maryam Shambayati, MD
Facility Name
Prisma Health
City
Greenville
State/Province
South Carolina
ZIP/Postal Code
29615
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Carrie Frost
First Name & Middle Initial & Last Name & Degree
Jonathan Markowitz, M.D.
Facility Name
GI For Kids, PLLC
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
32963
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Paul Patterson
Phone
865-560-0196
Email
PJPatterson@utmck.edu
First Name & Middle Initial & Last Name & Degree
Youhanna Al-Tawil, MD
Facility Name
Newco 3A Research,LLC DBA 3A Research
City
El Paso
State/Province
Texas
ZIP/Postal Code
79902
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Karen Salas
Phone
915-598-8888
Email
karen@3asites.com
First Name & Middle Initial & Last Name & Degree
Aldo Maspons, MD
Facility Name
Pediatric GI of El Paso, LLC
City
El Paso
State/Province
Texas
ZIP/Postal Code
79925
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Helen Gutierrez
Phone
915-615-7005
Email
helengtz@elppedsgi.com
First Name & Middle Initial & Last Name & Degree
Eduardo D. Rosas-Blum, MD
Facility Name
McGovern Medical School of UT Health
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nicole Fatheree
Phone
713-500-5563
Email
nicole.fatheree@uth.tmc.edu
First Name & Middle Initial & Last Name & Degree
Melissa Van Arsdall, MD

12. IPD Sharing Statement

Learn more about this trial

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

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