Congenital Sucrase-isomaltase Deficiency (CSID)CSID GPSNCT01914003
Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Prevalence Study (GPS)
An observational study of Congenital Sucrase-isomaltase Deficiency (CSID), run by QOL Medical, LLC at 19 sites in United States.
Trial summary
Congenital sucrose-isomaltase deficiency (CSID) is a rare, genetic disease in which mutations in the sucrose-isomaltase (SI) gene cause digestion problems of sucrose resulting in diarrhea and abdominal pain. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Congenital Sucrase-isomaltase Deficiency (CSID)
- How many people
- 53 participants
- Where
- 19 sitesUnited States
- Study dates
- May 2013to Jul 2015
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: 18 years of age or younger experiencing chronic, idiopathic diarrhea or abdominal pain for at least 4 weeks.
Who may be able to join
- Must be 18 years of age or younger.
- A primary clinical diagnosis of chronic idiopathic diarrhea or chronic abdominal pain for at least 4 weeks.
- English or Spanish speaking subjects and parent(s)/guardian only.
- Parental consent from one parent/guardian and also subject assent when appropriate based on individual IRB requirements.
Who cannot join
- Any condition(s) or finding(s) that in the opinion of the principal investigator suggests an alternative diagnosis for his/her gastrointestinal symptoms.
- Abdominal pain primarily related to constipation.
- Suspected gastrointestinal infectious disease.
- No current use of sacrosidase (Sucraid® Oral Solution).
- Known gastrointestinal disease such as celiac disease.
- Prior consumption of an investigational medication within the last 4 weeks.
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- Antibiotics in the last 2 weeks, and no history of viral gastroenteritis within that same period of time.
- Known Hepatitis B or C infection (positive HBsAg or HCV within 6 months of enrollment) or Subject-Pugh Class C liver disease of any cause, HIV infection, tuberculosis, Clostridia difficile co-infection, cancer or systemic infections.
- Severe neurologic impairment that would prevent them from reporting a history of abdominal pain.
- Receiving or received biologic therapies (including infliximab, adalimumab, natalizumab) within 3 months prior to or at enrollment.
- Present or past use of immune modulators therapy (e.g., azathioprine, 6MP, methotrexate).
- Planned or previous abdominal surgery (e.g., bowel resection).
- Subjects with severe, uncontrolled systemic diseases.
- Presence of clinical alarm signs, including hypotension, anemia requiring blood transfusions, altered mental status, or inability to tolerate food and/or fluids by mouth.
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
United States19 sites
Children's Center for Digestive Healthcare
Atlanta, Georgia
Children's Hospital of Colorado
Aurora, Colorado
Johns Hopkins Children's Center
Baltimore, Maryland
Massachusetts General Hospital
Boston, Massachusetts
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois
Nationwide Children's Hospital
Columbus, Ohio
Duke University Children's Hospital
Durham, North Carolina
Texas Children's Hospital
Houston, Texas
Riley Hospital for Children
Indianapolis, Indiana
University of Mississippi Medical Center
Jackson, Mississippi
Children's Mercy Hospital
Kansas City, Missouri
Children's Hospital Los Angeles
Los Angeles, California
Children's Hospital of Wisconsin
Milwaukee, Wisconsin
Morgan Stanley Children's Hospital
New York, New York
Children's Hospital and Research Center of Oakland
Oakland, California
Arnold Palmer Children's Hospital
Orlando, Florida
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania
Primary Children's Medical Center
Salt Lake City, Utah
Stony Brook University
Stony Brook, New York
About this study
Official title: A Multi-Center Study of the Prevalence of Known Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Variants and Functional Sucrase Activity by 13C-Sucrose Breath Test in Children With Chronic Diarrhea or Chronic Abdominal Pain
Congenital sucrose-isomaltase deficiency (CSID) is a rare, genetic disease in which mutations in the sucrose-isomaltase (SI) gene cause digestion problems of sucrose resulting in diarrhea and abdominal pain. Children with chronic, idiopathic diarrhea or abdominal pain will have their sucrose-isomaltase gene assessed for a panel of known CSID mutations to determine the prevalence of these mutations in an enriched population and also determine functional deficiency using a breath test.
- 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.
| Study group | Role | What they receive |
|---|---|---|
| CSID Mutations | Individual has one or more known CSID mutations. | |
| Control | Individual does not have any known CSID mutations. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of CSID Genetic Variants
Prevalence of CSID genetic variants in subjects 18 years of age or younger with a primary symptom of chronic idiopathic diarrhea or chronic abdominal pain without constipation.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2013 (estimated)
- Study startMay 2013
- Primary completionMay 2015
- Study completionJul 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Congenital Sucrase-isomaltase Deficiency (CSID)Studies 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 53 participants (actual)
Sponsor and investigators
- Lead sponsor
- QOL Medical, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Arnold Palmer Hospital for Children, Baylor College of Medicine, Nationwide Children's Hospital, Ann & Robert H Lurie Children's Hospital of Chicago, University of Mississippi Medical Center, Children's Hospital and Research Center at Oakland, Columbia University, Children's Hospital Los Angeles, Children's Hospital and Health System Foundation, Wisconsin, Children's Center for Digestive Healthcare, LLC, Massachusetts General Hospital, Duke University, Johns Hopkins University, Children's Hospital of Philadelphia, Children's Mercy Hospital Kansas City, Children's Hospital Colorado, Riley Children's Hospital, Primary Children's Hospital, State University of New York - Downstate Medical Center
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01914003
- Sponsor's protocol ID
- S2002
- Acronym
- CSID GPS
- Status last verified
- Aug 2016
- First submitted
- 22 Jul 2013
- Last updated
- 6 Nov 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center Study of the Prevalence of Known Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Variants and Functional Sucrase Activity by 13C-Sucrose Breath Test in Children With Chronic Diarrhea or Chronic Abdominal Pain
- ClinicalTrials.gov ID
- NCT01914003
- Acronym / sponsor ID
- CSID GPS / S2002
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 53 participants (actual)
- Lead sponsor
- QOL Medical, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Arnold Palmer Hospital for Children, Baylor College of Medicine, Nationwide Children's Hospital, Ann & Robert H Lurie Children's Hospital of Chicago, University of Mississippi Medical Center, Children's Hospital and Research Center at Oakland, Columbia University, Children's Hospital Los Angeles, Children's Hospital and Health System Foundation, Wisconsin, Children's Center for Digestive Healthcare, LLC, Massachusetts General Hospital, Duke University, Johns Hopkins University, Children's Hospital of Philadelphia, Children's Mercy Hospital Kansas City, Children's Hospital Colorado, Riley Children's Hospital, Primary Children's Hospital, State University of New York - Downstate Medical Center
- Study start
- May 2013
- Primary completion
- May 2015 (actual)
- Study completion
- Jul 2015 (actual)
- First posted
- 1 Aug 2013 (estimated)
- Results
- Posted 10 Oct 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Congenital sucrose-isomaltase deficiency (CSID) is a rare, genetic disease in which mutations in the sucrose-isomaltase (SI) gene cause digestion problems of sucrose resulting in diarrhea and abdominal pain. Children with chronic, idiopathic diarrhea or abdominal pain will have their sucrose-isomaltase gene assessed for a panel of known CSID mutations to determine the prevalence of these mutations in an enriched population and also determine functional deficiency using a breath test.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CSID Mutations | – | –Individual has one or more known CSID mutations. |
| Control | – | –Individual does not have any known CSID mutations. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- 18 years of age or younger experiencing chronic, idiopathic diarrhea or abdominal pain for at least 4 weeks.
Key inclusion criteria (4 listed)
- Must be 18 years of age or younger.
- A primary clinical diagnosis of chronic idiopathic diarrhea or chronic abdominal pain for at least 4 weeks.
- English or Spanish speaking subjects and parent(s)/guardian only.
- Parental consent from one parent/guardian and also subject assent when appropriate based on individual IRB requirements.
Key exclusion criteria (14 listed)
- Any condition(s) or finding(s) that in the opinion of the principal investigator suggests an alternative diagnosis for his/her gastrointestinal symptoms.
- Abdominal pain primarily related to constipation.
- Suspected gastrointestinal infectious disease.
- No current use of sacrosidase (Sucraid® Oral Solution).
- Known gastrointestinal disease such as celiac disease.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Prevalence of CSID Genetic Variants
Time frame: 1 year
Prevalence of CSID genetic variants in subjects 18 years of age or younger with a primary symptom of chronic idiopathic diarrhea or chronic abdominal pain without constipation.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Study overviewLink provided by the sponsor.
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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.
The record lists no contact. Use the official record to reach the sponsor.
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Sucrase-isomaltase Deficiency (CSID), run by QOL Medical, LLC at 19 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 4 inclusion and 14 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: finished, as of the sponsor's last update on 6 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Prevalence of CSID Genetic Variants (time frame: 1 year). 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 6 Nov 2017.