Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis

This study is currently recruiting participants.

Verified by FDA Office of Orphan Products Development, July 1998

First Received: October 18, 1999 Last Updated: June 23, 2005 History of Changes

Sponsor:	FDA Office of Orphan Products Development
Collaborator:	Children's Hospital Medical Center, Cincinnati
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00004441

Purpose

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:	Treatment, Efficacy Study

Resource links provided by NLM:

Genetics Home Reference related topics: cystic fibrosis

MedlinePlus related topics: Cystic Fibrosis

Drug Information available for: Tauroursodeoxycholic acid

U.S. FDA Resources

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

Estimated Enrollment:	39
Study Start Date:	September 1997

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.

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

PROTOCOL ENTRY CRITERIA:

--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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004441

Locations

United States, Colorado
Children's Hospital of Denver	Recruiting
Denver, Colorado, United States, 80218
Contact: Michael Narkowicz 303-861-6669
United States, Ohio
Children's Hospital Medical Center - Cincinnati	Recruiting
Cincinnati, Ohio, United States, 45229-3039
Contact: Kenneth Setchell 513-636-4548
Italy
University of Milan	Recruiting
Milan, Italy, 20122
Contact: Carla Colombo 2-8-912-9975

Sponsors and Collaborators

FDA Office of Orphan Products Development

Children's Hospital Medical Center, Cincinnati

Investigators

Study Chair:

Kenneth Setchell

Children's Hospital Medical Center, Cincinnati

More Information

No publications provided

Study ID Numbers:	199/13439, CHMC-C-001439, CHMC-C-96-1-8, CHMC-C-FDR001439-01
Study First Received:	October 18, 1999
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004441 History of Changes
Health Authority:	United States: Federal Government

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:

Anti-Infective Agents
Taurochenodeoxycholic Acid
Fibrosis
Tauroursodeoxycholic acid
Gastrointestinal Agents
Antiviral Agents
Pharmacologic Actions
Digestive System Diseases
Pathologic Processes

Cystic Fibrosis
Respiratory Tract Diseases
Genetic Diseases, Inborn
Therapeutic Uses
Lung Diseases
Cholagogues and Choleretics
Pancreatic Diseases
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on November 09, 2009