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Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis

This study has been completed.
Information provided by:
FDA Office of Orphan Products Development Identifier:
First received: October 18, 1999
Last updated: March 24, 2015
Last verified: July 1998

OBJECTIVES: I. Determine the optimum dose of tauroursodeoxycholic acid (TUDCA) required to achieve maximal bioavailability for patients with cystic fibrosis-associated liver disease.

II. Compare optimized doses of TUDCA with ursodiol (ursodeoxycholic acid; UDCA) for effects on biliary bile acid composition and metabolism, serum biochemistries, fat absorption, and fat-soluble vitamin status in these patients.

Condition Intervention
Cystic Fibrosis Drug: tauroursodeoxycholic acid Drug: ursodiol

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

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

Estimated Enrollment: 39
Study Start Date: September 1997
Estimated Study Completion Date: September 2001
Detailed Description:

PROTOCOL OUTLINE: Objective I: This part of the study is a dose-response study to determine the optimal dose of tauroursodeoxycholic acid (TUDCA). Twenty-four patients are randomized to receive one of three different doses of TUDCA for 3 months.

Objective II: This part of the study is a double-blind crossover study to compare optimized doses of TUDCA with optimized doses of ursodiol in 15 patients stratified according to age (less than 10 vs 10-20 vs more than 20 years). Patients are randomized to receive either TUDCA or ursodiol orally for an initial 3 month period, followed by a 3 month washout period in which no drug is administered. Patients then receive the alternate drug for 3 months.

Completion date provided represents the completion date of the grant per OOPD records


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics--

  • Cystic fibrosis-associated liver disease, defined by at least one of the following criteria: (1) Documented increase in serum concentrations of any of the liver enzymes (at least once in the preceding year) ALT at least twice normal AST at least 1.5 times normal Alkaline phosphatase at least 1.5 times normal GGT at least 1.5 times normal (2) Persistent hepatomegaly of more than 6 months duration defined by percussed liver span greater than 1 SEM for age (3) Splenomegaly, defined as a palpable spleen greater than 2.0 cm below the left costal margin (4) Abnormalities of ultrasound scan (increased size, dishomogeneous echogenicity, nodular liver, irregular margins, splenomegaly) within 6 months prior to study entry
  • Patients enrolled in the first part of the study (objective I) are eligible to participate in the second part (objective II)

--Prior/Concurrent Therapy--

  • At least 3 months since prior ursodiol
  • At least 3 months since treatment with drug with choleretic properties or effects that influence bile acid metabolism

--Patient Characteristics--

  • Hepatic: No decompensated cirrhosis No hepatic neoplasm or cholelithiasis
  • Pulmonary: No significantly impaired pulmonary function with FEV1 less than 50%
  • Other: At least 15 kg body weight No severely compromised clinical or nutritional state
  Contacts and Locations
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Please refer to this study by its identifier: NCT00004441

United States, Colorado
Children's Hospital of Denver
Denver, Colorado, United States, 80218
United States, Ohio
Children's Hospital Medical Center - Cincinnati
Cincinnati, Ohio, United States, 45229-3039
University of Milan
Milan, Italy, 20122
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Study Chair: Kenneth Setchell Children's Hospital Medical Center, Cincinnati
  More Information Identifier: NCT00004441     History of Changes
Other Study ID Numbers: 199/13439
Study First Received: October 18, 1999
Last Updated: March 24, 2015

Keywords provided by FDA Office of Orphan Products Development:
cardiovascular and respiratory diseases
cystic fibrosis
genetic diseases and dysmorphic syndromes
rare disease

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Tauroursodeoxycholic acid
Taurochenodeoxycholic Acid
Antiviral Agents
Anti-Infective Agents
Cholagogues and Choleretics
Gastrointestinal Agents processed this record on August 18, 2017