Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis

NCT00004441 | Completed

• 4 other identifiers: 199/13439CHMC-C-001439CHMC-C-96-1-8CHMC-C-FDR001439-01
• Study Type: interventional
• Target: 39
• Locations: 2 countries
• Sites: 3

Brief Summary

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.

Conditions

Cystic Fibrosis

Keywords

cardiovascular and respiratory diseases; cystic fibrosis; genetic diseases and dysmorphic syndromes; rare disease

Interventions

tauroursodeoxycholic acid DRUG

ursodiol DRUG

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (3)

Children's Hospital of Denver

Denver, Colorado, 80218, United States

Location

Children's Hospital Medical Center - Cincinnati

Cincinnati, Ohio, 45229-3039, United States

Location

University of Milan

Milan, 20122, Italy

Location

MeSH Terms

Conditions

Cystic Fibrosis; Respiratory Tract Diseases; Genetic Diseases, Inborn; Rare Diseases

Interventions

ursodoxicoltaurine; Ursodeoxycholic Acid

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Deoxycholic Acid; Cholic Acids; Bile Acids and Salts; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cholanes

Study Officials

• Kenneth Setchell

  Children's Hospital Medical Center, Cincinnati

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: October 18, 1999
• First Posted: October 19, 1999
• Study Start: September 1, 1997
• Study Completion: September 1, 2001
• Last Updated: March 25, 2015

Record last verified: 1998-07

Locations

US (2); IT (1)