Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis
tauroursodeoxycholic acid
+ ursodiol
Maladies du système digestif+9
+ Maladies du nouveau-né
+ Maladies pulmonaires
Étude thérapeutique
Résumé
Date de début de l'étude : 1 septembre 1997
Date à laquelle le premier participant a commencé l'étude.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
Protocole
Cette section fournit des détails sur le plan de l'étude, y compris la manière dont l'étude est conçue et ce qu'elle évalue.39 participants à inclure
Nombre total de participants que l'essai clinique vise à recruter.Traitement
Éligibilité
Les chercheurs recherchent des patients correspondant à une certaine description appelée critères d'éligibilité : état de santé général ou traitements antérieurs du patient.Tout sexe
Le sexe biologique des participants éligibles à s'inscrire.Volontaires sains non autorisés
Indique si les individus en bonne santé et ne présentant pas la condition étudiée peuvent participer.Conditions
Pathologie
Critères
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
Centres d'étude
Ce sont les hôpitaux, cliniques ou centres de recherche où l'essai est conduit. Vous pouvez trouver le site le plus proche de vous ainsi que son statut.Cette étude comporte 3 sites
Children's Hospital of Denver
Denver, United StatesOuvrir Children's Hospital of Denver dans Google MapsChildren's Hospital Medical Center - Cincinnati
Cincinnati, United StatesUniversity of Milan
Milan, Italy