Clinical trial NCT05480761: 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
Overall status: TERMINATED
Phase: PHASE4
Conditions: Congenital Sucrase-Isomaltase Deficiency
CSID
Sucrase Isomaltase Deficiency
Keywords: www.tummyachestudy.com
Lead sponsor: QOL Medical, LLC
Sponsor class: INDUSTRY
First posted: 2022-07-29
Enrollment: 312
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Effects of Sucraid® treatment on gastrointestinal symptoms in subjects with low, moderate, and normal disaccharidase assay sucrase levels from an esophagogastroduodenoscopy (EGD).
timeFrame: Post 7-day run-in period plus 7-day treatment period.
Secondary outcomes:
measure: Explore the relationship between known genetic CSID mutations and sucrase activities via (EGD) disaccharidase assay (low, moderate, and normal).
timeFrame: Up to 2 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Coral Gables
country: United States
facility: Kidz Medical
state: Florida
city: Orlando
country: United States
facility: Orlando Health
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Atlanta
country: United States
facility: Center for Digestive Health Care, LLC
state: Georgia
city: Baltimore
country: United States
facility: University of Maryland Baltimore
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Flowood
country: United States
facility: GI Associates
state: Mississippi
city: Flowood
country: United States
facility: Happy Tummies
state: Mississippi
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Morristown
country: United States
facility: Atlantic Health System
state: New Jersey
city: Buffalo
country: United States
facility: WNY Pediatric Gastroenterology
state: New York
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Oklahoma City
country: United States
facility: Measurable Outcomes Research
state: Oklahoma
city: Greenville
country: United States
facility: Prisma Health
state: South Carolina
city: Knoxville
country: United States
facility: GI For Kids, PLLC
state: Tennessee
city: El Paso
country: United States
facility: Newco 3A Research,LLC DBA 3A Research
state: Texas
city: El Paso
country: United States
facility: Pediatric GI of El Paso, LLC
state: Texas
city: Houston
country: United States
facility: McGovern Medical School of UT Health
state: Texas
Study officials:
affiliation: QOL Medical
name: Weng Tao, M.D., Ph. D
role: PRINCIPAL_INVESTIGATOR
References: