Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis
tauroursodeoxycholic acid
+ ursodiol
Digestive System Diseases+9
+ Infant, Newborn, Diseases
+ Lung Diseases
Treatment Study
Summary
Study start date: September 1, 1997
Actual date on which the first participant was enrolled.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
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.39 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
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
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 3 locations
Children's Hospital Medical Center - Cincinnati
Cincinnati, United StatesUniversity of Milan
Milan, Italy