Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis - Full Text View

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

Sponsors and Collaborators:	FDA Office of Orphan Products Development 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

Genetics Home Reference related topics:

cystic fibrosis

MedlinePlus related topics:

Cystic Fibrosis

Drug Information available for:

Tauroursodeoxycholic acid Ursodeoxycholic acid

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Efficacy Study

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

Study ID Numbers:	199/13439, CHMC-C-001439, CHMC-C-96-1-8, CHMC-C-FDR001439-01
First Received:	October 18, 1999
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004441
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

Study placed in the following topic categories:

Taurochenodeoxycholic Acid

Fibrosis

Respiration Disorders

Tauroursodeoxycholic acid

Rare Diseases

Ursodeoxycholic Acid

Digestive System Diseases

Cystic Fibrosis

Respiratory Tract Diseases

Genetic Diseases, Inborn

Lung Diseases

Pancreatic Diseases

Infant, Newborn, Diseases

Cystic fibrosis

Additional relevant MeSH terms:

Anti-Infective Agents

Pathologic Processes

Cholagogues and Choleretics

Therapeutic Uses

Gastrointestinal Agents

Antiviral Agents

Pharmacologic Actions

ClinicalTrials.gov processed this record on November 20, 2008