Status:

COMPLETED

Congenital Sucrase-Isomaltase Deficiency (CSID) Genetic Prevalence Study (GPS)

Lead Sponsor:

QOL Medical, LLC

Collaborating Sponsors:

Arnold Palmer Hospital for Children

Baylor College of Medicine

Conditions:

Congenital Sucrase-isomaltase Deficiency (CSID)

Eligibility:

All Genders

Up to 18 years

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 abdomina...

Eligibility Criteria

Inclusion

  • 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.

Exclusion

  • 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.

Key Trial Info

Start Date :

May 1 2013

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

July 1 2015

Estimated Enrollment :

53 Patients enrolled

Trial Details

Trial ID

NCT01914003

Start Date

May 1 2013

End Date

July 1 2015

Last Update

November 6 2017

Active Locations (19)

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Page 1 of 5 (19 locations)

1

Children's Hospital Los Angeles

Los Angeles, California, United States

2

Children's Hospital and Research Center of Oakland

Oakland, California, United States

3

Children's Hospital of Colorado

Aurora, Colorado, United States

4

Arnold Palmer Children's Hospital

Orlando, Florida, United States