Congenital Sucrase-Isomaltase DeficiencyPhase 4NCT05480761
7-Day Trial of Sucraid for Alleviating CSID Symptoms in Subjects With Low, Moderate, and Normal Sucrase Levels
A Phase 4 study testing Sucraid (sacrosidase) Oral Solution 8500 IU/mL in people with Congenital Sucrase-Isomaltase Deficiency and CSID, run by QOL Medical, LLC at 20 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 months to 17 years
- Conditions the study is for
- Congenital Sucrase-Isomaltase Deficiency, CSID, Sucrase Isomaltase Deficiency
- What is being tested?
- Sucraid (sacrosidase) Oral Solution 8500 IU/mL
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 312 participants
- Where
- 20 sitesUnited States
- Study dates
- 1 Aug 2022to 18 Sep 2024
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
United States20 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
Center for Digestive Health Care, LLC
Atlanta, Georgia, 30342
University of Maryland Baltimore
Baltimore, Maryland, 21201
Johns Hopkins University
Baltimore, Maryland, 21205
WNY Pediatric Gastroenterology
Buffalo, New York, 14225
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Kidz Medical
Coral Gables, Florida, 33146
Newco 3A Research,LLC DBA 3A Research
El Paso, Texas, 79902
Pediatric GI of El Paso, LLC
El Paso, Texas, 79925
GI Associates
Flowood, Mississippi, 39232
Happy Tummies
Flowood, Mississippi, 39232
Prisma Health
Greenville, South Carolina, 29615
McGovern Medical School of UT Health
Houston, Texas, 77030
Children's Mercy Hospital
Kansas City, Missouri, 64108
GI For Kids, PLLC
Knoxville, Tennessee, 32963
Children's Hospital Los Angeles
Los Angeles, California, 90027
Atlantic Health System
Morristown, New Jersey, 07960
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Measurable Outcomes Research
Oklahoma City, Oklahoma, 73112
Orlando Health
Orlando, Florida, 32806
About this study
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
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).
Read the 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.
- Being tested
- Sucraid (sacrosidase) Oral Solution 8500 IU/mL
- Keywords
- www.tummyachestudy.com
- Medical subject headings
- Sucrase-isomaltase deficiency, congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sucraid (sacrosidase) Oral Solution 8500 IU/mLBiological
Also called Sucraid, Sacrosidase
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.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm Open Labeled Commercial Sucraid | Experimental | All subjects will complete a 7-day treatment period of open-labeled FDA approved commercial Sucraid. Receives: Biological: Sucraid (sacrosidase) Oral Solution 8500 IU/mL |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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).
The number and percentage of subjects with genetic CSID mutations will also be examined in low, moderate, and normal sucrase study group.
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2022
- Study start1 Aug 2022
- Primary completion18 Sep 2024
- Study completion18 Sep 2024
Why it stopped: Lack of subject enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital Sucrase-Isomaltase DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 312 participants (actual)
Sponsor and investigators
- Lead sponsor
- QOL Medical, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Weng Tao, M.D., Ph. D, Principal investigator, QOL Medical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05480761
- Sponsor's protocol ID
- SSDXP-13
- Status last verified
- Mar 2023
- First submitted
- 28 Jun 2022
- Last updated
- 20 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05480761
- Acronym / sponsor ID
- SSDXP-13
- Keywords
- www.tummyachestudy.com
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of subject enrollment
- Enrolment
- 312 participants (actual)
- Lead sponsor
- QOL Medical, LLCIndustry (pharma, biotech, devices)
- Study start
- 1 Aug 2022 (actual)
- Primary completion
- 18 Sep 2024 (actual)
- Study completion
- 18 Sep 2024 (actual)
- First posted
- 29 Jul 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm Open Labeled Commercial Sucraid | Experimental | Biological: Sucraid (sacrosidase) Oral Solution 8500 IU/mLAll subjects will complete a 7-day treatment period of open-labeled FDA approved commercial Sucraid. |
Who may qualify
- Ages
- 6 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- 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.
Key exclusion criteria (9 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sucraid (sacrosidase) Oral Solution 8500 IU/mLAlso: Sucraid, SacrosidaseSucraid 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. | Biological | Single Arm Open Labeled Commercial Sucraid |
Primary outcome
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.
1 secondary outcome
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
Results and safety
The study was stopped early (reason given: Lack of subject enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Weng Tao, M.D., Ph. D principal investigator
QOL Medical
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Sucraid (sacrosidase) Oral Solution 8500 IU/mL in people with Congenital Sucrase-Isomaltase Deficiency and CSID, run by QOL Medical, LLC at 20 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 20 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Lack of subject enrollment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Mar 2025.