URSODEOXYCHOLIC ACID for Hepatorenal polycystic diseases

Inclusion criteria

• {"criterion_text":"- Age: 18 - 80 years (inclusive).\n- Diagnosed liver or hepatorenal polycystic disease and total liver volume 2000-5000 mL.\n- PLD defined as >10 liver cysts and PKD defined as: -\tAt least 2 kidney cysts or 1 cyst in each kidney in patients younger than 30 years. -\tAt least 2 cysts in each kidney in patients aged 30-59 years old. -\tAt least 4 cysts in each kidney in patients aged 60 years or older.\n- Not be candidates for intervention (aspiration or surgical intervention) at the time of inclusion."}

Exclusion criteria

• {"criterion_text":"- TLV >5000 mL\n- Renal dysfunction (MDRD- GFR ≤ 30 ml/min/1.73m2).\n- Use of hormonal contraceptives or estrogen supplements in the previous 6 months.\n- Inclusion in another clinical trial while participating in this study.\n- Pregnant or lactating women, or subjects of childbearing potential who are not using an effective method of contraception.\n- History or other evidence of serious illness or any other condition that would make the patient, in the opinion of the investigator, unsuitable for the study.\n- Gastric or duodenal ulcers.\n- Use of drugs that may interact with UDCA, such as cholestyramine or aluminum hydroxide.\n- Treatment with UDCA in the previous 6 months\n- Chronic liver disease unrelated to PLD.\n- Intervention (aspiration or surgical intervention) in the previous 6 months.\n- Treatment with somatostatin analogues in the previous 6 months.\n- Hypersensitivity to UDCA or patients with galactose intolerance or malabsorption, or lactase deficiency.\n- Acute cholecystitis or frequent attacks of biliary colic.\n- Treatment with tolvaptan in the previous 6 months."}

Primary endpoints

• {"endpoint_text":"- 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.","definition_or_measurement_approach":"Absolute and percentage change in total liver cyst volume (TLCV) measured at baseline and at end of treatment and compared between the UDCA and placebo groups."}

Secondary endpoints

• {"endpoint_text":"- Liver volume: absolute and proportional change in liver volume (TLV/hTLV).\n- Renal volume: absolute and proportional change in renal volume (TKV/hTKV).\n- Renal function: glomerular filtration rate (GFR), serum creatinine, indication for dialysis or renal transplantation.\n- Laboratory values: haematology, biochemistry, hormones, coagulation, liver profile (ALT, AST, γGT, ALP, Bi) and CA-19-9.\n- Symptomatology: to be assessed at each visit to the centre using the PLD-Q questionnaire.\n- Quality of life: SF-36 questionnaire.\n- Safety: occurrence of serious adverse events related to the medication and any events occurring during the patient's participation in the trial.\n- Genetic profile: centralized analysis of genes described for the disease.\n- (Exploratory endpoint) - Demographic variables (age, sex).\n- (Exploratory endpoint) - Medical history.\n- (Exploratory endpoint) -Concomitant medication and adherence to treatment.\n- (Exploratory endopoint) - Physical examination (body weight, height, blood pressure, heart rate).\n- (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.\n- (Exploratory endopoint) - Association analysis between genotype and clinical response to treatment.\n- (Exploratory endopoint) -Treatment response markers based on proteins, RNA, and/or circulating metabolites, among others (liquid biopsy).\n- (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.","definition_or_measurement_approach":"Definitions and measurement approaches are provided within each endpoint description (e.g., absolute and proportional change in volume measures, GFR and serum creatinine for renal function, PLD-Q and SF-36 questionnaires for symptoms and QoL, centralised genetic analysis for genetic profile)."}

Other endpoints

• {"endpoint_text":"- (Exploratory endpoint) - Demographic variables (age, sex).\n- (Exploratory endpoint) - Medical history.\n- (Exploratory endpoint) -Concomitant medication and adherence to treatment.\n- (Exploratory endopoint) - Physical examination (body weight, height, blood pressure, heart rate).\n- (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.\n- (Exploratory endopoint) - Association analysis between genotype and clinical response to treatment.\n- (Exploratory endopoint) -Treatment response markers based on proteins, RNA, and/or circulating metabolites, among others (liquid biopsy).\n- (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.","definition_or_measurement_approach":"Exploratory endpoints measured as described in endpoint text: demographics and medical history recorded; concomitant medication and adherence recorded; physical exam measures; imaging-derived cyst and organ volumes at baseline, 1 year and end of treatment; central genotype-phenotype association analyses and biomarker (protein/RNA/metabolite) assays (liquid biopsy) correlated with volumetric and clinical outcomes."}

Spain

Earliest CTIS Part Ii Submission Date

09-01-2026

Latest Decision Or Authorization Date

02-03-2026

Processing Time Days

52

Number Of Sites

1

Number Of Participants

138

Primary sponsor

Full Name

Asociacion Instituto Biogipuzkoa

Organisation Type

Patient organisation/association

Country Of Registered Address

Spain