NCT01914003

Brief 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. 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
53

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started May 2013

Typical duration for all trials

Geographic Reach
1 country

19 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2013

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

July 22, 2013

Completed
10 days until next milestone

First Posted

Study publicly available on registry

August 1, 2013

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2015

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2015

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

October 10, 2016

Completed
Last Updated

November 6, 2017

Status Verified

August 1, 2016

Enrollment Period

2 years

First QC Date

July 22, 2013

Results QC Date

July 7, 2016

Last Update Submit

October 2, 2017

Conditions

Congenital Sucrase-isomaltase Deficiency (CSID)

Outcome Measures

Primary Outcomes (1)

  • 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.

    1 year

Study Arms (2)

CSID Mutations

Individual has one or more known CSID mutations.

Control

Individual does not have any known CSID mutations.

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

18 years of age or younger experiencing chronic, idiopathic diarrhea or abdominal pain for at least 4 weeks.

You may qualify if:

  • 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.

You may not qualify if:

  • 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.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (19)

Children's Hospital Los Angeles

Los Angeles, California, United States

Location

Children's Hospital and Research Center of Oakland

Oakland, California, United States

Location

Children's Hospital of Colorado

Aurora, Colorado, United States

Location

Arnold Palmer Children's Hospital

Orlando, Florida, United States

Location

Children's Center for Digestive Healthcare

Atlanta, Georgia, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, United States

Location

Johns Hopkins Children's Center

Baltimore, Maryland, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, United States

Location

Morgan Stanley Children's Hospital

New York, New York, United States

Location

Stony Brook University

Stony Brook, New York, United States

Location

Duke University Children's Hospital

Durham, North Carolina, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Location

Texas Children's Hospital

Houston, Texas, United States

Location

Primary Children's Medical Center

Salt Lake City, Utah, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Location

Related Links

MeSH Terms

Conditions

Sucrase-isomaltase deficiency, congenital

Results Point of Contact

Title
Heather Smith, Director of One Patient Services
Organization
QOL Medical, LLC
hsmith@onepatientservices.com
919-832-4949

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 22, 2013

First Posted

August 1, 2013

Study Start

May 1, 2013

Primary Completion

May 1, 2015

Study Completion

July 1, 2015

Last Updated

November 6, 2017

Results First Posted

October 10, 2016

Record last verified: 2016-08

Locations

US(19)