Efficacy and safety of Ursodeoxycholic Acid as a new therapy for the treatment of hepatorenal polycystic diseases, genetic study and determination of prognostic biomarkers.

Condition studied

Hepatorenal polycystic diseases

Investigational medicinal product(s)

Eligibility

Primary endpoint

The primary endpoint is the absolute and percentage change in total liver cyst volume (TLCV) at the beginning and the end of treatment between the two groups.

Endpoint detail

Liver volume: absolute and proportional change in liver volume (TLV/hTLV)., Renal volume: absolute and proportional change in renal volume (TKV/hTKV)., Renal function: glomerular filtration rate (GFR), serum creatinine, indication for dialysis or renal transplantation., Laboratory values: haematology, biochemistry, hormones, coagulation, liver profile (ALT, AST, γGT, ALP, Bi) and CA-19-9., Symptomatology: to be assessed at each visit to the centre using the PLD-Q questionnaire., Quality of life: SF-36 questionnaire., Safety: occurrence of serious adverse events related to the medication and any events occurring during the patient's participation in the trial., Genetic profile: centralized analysis of genes described for the disease., (Exploratory endpoint) - Demographic variables (age, sex)., (Exploratory endpoint) - Medical history., (Exploratory endpoint) -Concomitant medication and adherence to treatment., (Exploratory endopoint) - Physical examination (body weight, height, blood pressure, heart rate)., (Exploratory endopoint) - Kidney cyst volume: absolute and proportional change in renal cyst volume change (TKCV), at the baseline visit, at one year and at the end of treatment., (Exploratory endopoint) - Association analysis between genotype and clinical response to treatment., (Exploratory endopoint) -Treatment response markers based on proteins, RNA, and/or circulating metabolites, among others (liquid biopsy)., (Exploratory endopoint) - Correlation of biomarker levels with: - TLV / hTLV, TLCV and TKV / hTKV and its progression over time, - Clinical response - Liver and kidney function parameters.