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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Asociacion Instituto Biogipuzkoa

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Decision date (initial)

2026-03-02

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the therapeutic efficacy of UDCA versus placebo in patients with PLD by decreasing the total liver cyst volume (TLCV) growth (prevention of disease progression).

Secondary objectives 12

1. To assess the change in total liver volume (TLV / hTLV) and/or growth overtime.
2. To assess the change in total kidney volume (TKV / hTLV) and/or growth overtime.
3. To analyze whether UDCA produces improvement in renal function or stabilises it.
4. To assess the effect of treatment on cholestasis enzymes and liver function.
5. Determine whether UDCA improves patients' symptomatology
6. To assess the impact on quality of life of UDCA treatment.
7. To assess the safety and tolerability of UDCA as a treatment for PLD
8. Determine disease progression according to the genes involved and the mutations present in the development of PLD.
9. (Exploratory objectives) - To assess the change in total kidney cystic volume (TKCV) and/or growth overtime.
10. (Exploratory objectives) - To investigate the association between genetic variants in PLD and clinical response to treatment.
11. (Exploratory objectives) - To explore biomarkers as predictors of liver disease progression and/or treatment response.
12. (Exploratory objectives) - To explore variability in treatment response through subgroup analyses.

Conditions and MedDRA coding

Hepatorenal polycystic diseases

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

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

Exclusion criteria 15

1. TLV >5000 mL
2. Renal dysfunction (MDRD- GFR ≤ 30 ml/min/1.73m2).
3. Use of hormonal contraceptives or estrogen supplements in the previous 6 months.
4. Inclusion in another clinical trial while participating in this study.
5. Pregnant or lactating women, or subjects of childbearing potential who are not using an effective method of contraception.
6. 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.
7. Gastric or duodenal ulcers.
8. Use of drugs that may interact with UDCA, such as cholestyramine or aluminum hydroxide.
9. Treatment with UDCA in the previous 6 months
10. Chronic liver disease unrelated to PLD.
11. Intervention (aspiration or surgical intervention) in the previous 6 months.
12. Treatment with somatostatin analogues in the previous 6 months.
13. Hypersensitivity to UDCA or patients with galactose intolerance or malabsorption, or lactase deficiency.
14. Acute cholecystitis or frequent attacks of biliary colic.
15. Treatment with tolvaptan in the previous 6 months.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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.

Secondary endpoints 16

1. Liver volume: absolute and proportional change in liver volume (TLV/hTLV).
2. Renal volume: absolute and proportional change in renal volume (TKV/hTKV).
3. Renal function: glomerular filtration rate (GFR), serum creatinine, indication for dialysis or renal transplantation.
4. Laboratory values: haematology, biochemistry, hormones, coagulation, liver profile (ALT, AST, γGT, ALP, Bi) and CA-19-9.
5. Symptomatology: to be assessed at each visit to the centre using the PLD-Q questionnaire.
6. Quality of life: SF-36 questionnaire.
7. Safety: occurrence of serious adverse events related to the medication and any events occurring during the patient's participation in the trial.
8. Genetic profile: centralized analysis of genes described for the disease.
9. (Exploratory endpoint) - Demographic variables (age, sex).
10. (Exploratory endpoint) - Medical history.
11. (Exploratory endpoint) -Concomitant medication and adherence to treatment.
12. (Exploratory endopoint) - Physical examination (body weight, height, blood pressure, heart rate).
13. (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.
14. (Exploratory endopoint) - Association analysis between genotype and clinical response to treatment.
15. (Exploratory endopoint) -Treatment response markers based on proteins, RNA, and/or circulating metabolites, among others (liquid biopsy).
16. (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.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Asociacion Instituto Biogipuzkoa

Sponsor organisation

Asociacion Instituto Biogipuzkoa

Address

Pasealeku Doct. Begiristain Sn

City

Donostia

Postcode

20014

Country

Spain

Scientific contact point

Organisation

Asociacion Instituto Biogipuzkoa

Contact name

Unidad de Investigación Clínica

Public contact point

Organisation

Asociacion Instituto Biogipuzkoa

Contact name

Unidad de Investigación Clínica

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 9 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications