Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Randomized Study of Tauroursodeoxycholic Acid in Prophylactic Therapy of Total Parenteral Nutrition Associated Cholestasis in Infants

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 1998 by FDA Office of Orphan Products Development.
Recruitment status was  Recruiting
Children's Hospital Medical Center, Cincinnati
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: June 23, 2005
Last verified: August 1998

OBJECTIVES: I. Determine whether infants treated with tauroursodeoxycholic acid (TUDCA) have a lower peak direct bilirubin, ALT, AST, glutamyltranspeptidase levels and a reduced duration of cholestasis compared to the nontreatment arm.

II. Determine the significance of lower birth weight and longer duration of total parenteral nutrition (TPN) on increasing risk of TPN associated cholestasis and increasing benefit from TUDCA therapy.

III. Determine whether TUDCA therapy leads to significant reduction in the appearance of biliary tract sludge and/or stone formation in these infants.

IV. Determine whether TUDCA therapy leads to reduced urinary excretion of potentially hepatotoxic bile acids as compared to the untreated arm matched for birth weight and duration of TPN.

Condition Intervention
Drug: tauroursodeoxycholic acid

Study Type: Interventional
Study Design: Allocation: Randomized
Primary Purpose: Treatment

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment: 50
Study Start Date: June 1998
Detailed Description:

PROTOCOL OUTLINE: This is a randomized study. Patients are stratified by birth weight.

Patients are randomized in pairs by birth weight to receive either a placebo in arm I or tauroursodeoxycholic acid (TUDCA) in arm II. TUDCA is administered by mouth, nasogastric tube, or gastrostomy tube twice daily. After 2 weeks of therapy, a bile sample is obtained via a duodenal tube. An ultrasound examination of the liver and biliary tract is performed after 2 weeks and every 3 weeks thereafter until discontinuation of therapy or until presence of biliary tract sludge is noted on 2 consecutive examinations.


Ages Eligible for Study:   up to 20 Days
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


--Disease Characteristics--

  • Hospitalized infants who are anticipated to require total parenteral nutrition for greater than 2 weeks and have: Major gastrointestinal anomalies (gastroschisis, ruptured omphalocele) OR Resection (necrotizing enterocolitis, volvulus)
  • No evidence of biliary tract abnormalities
  • No evidence of other forms of cholestatic liver disease

--Patient Characteristics--

  • Renal: No life threatening renal disease
  • Cardiovascular: No life threatening cardiovascular disease
  • Other: No multiple congenital abnormalities
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00004410

United States, Minnesota
Children's Hospitals and Clinics - Minneapolis Recruiting
Minneapolis, Minnesota, United States, 55404
Contact: Robert Couser    612-813-5913      
United States, Mississippi
University of Mississippi Medical Center Recruiting
Jackson, Mississippi, United States, 39216-4505
Contact: Phil Rhodes    601-984-5590      
United States, Ohio
Children's Hospital Medical Center - Cincinnati Recruiting
Cincinnati, Ohio, United States, 45229-3039
Contact: James Heubi    513-636-8046      
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Study Chair: James Heubi Children's Hospital Medical Center, Cincinnati
  More Information

No publications provided Identifier: NCT00004410     History of Changes
Other Study ID Numbers: 199/13299, CHMC-C-95-9-9, CHMC-C-CRC-473, CHMC-C-FDR001277
Study First Received: October 18, 1999
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:
gastrointestinal disorders
rare disease

Additional relevant MeSH terms:
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Taurochenodeoxycholic Acid
Tauroursodeoxycholic acid
Anti-Infective Agents
Antiviral Agents
Cholagogues and Choleretics
Gastrointestinal Agents
Pharmacologic Actions
Therapeutic Uses processed this record on March 01, 2015