Lanreotide Autogel in the Treatment of Symptomatic Polycystic Liver Disease

Study Overview
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Brief Summary
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An open-label, Phase II clinical study to evaluate the efficacy and safety of lanreotide autogel 90mg every 4 weeks in the treatment of symptomatic polycystic liver disease, including a dose escalation at month 6 to lanreotide autogel 120mg for non responders.

Official Title
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An Open-label, Phase II Clinical Study to Evaluate the Efficacy and Safety of Lanreotide Autogel 90mg Every 4 Weeks in the Treatment of Symptomatic Polycystic Liver Disease, Including a Dose Escalation at Month 6 to Lanreotide Autogel 120mg for Non Responders

Conditions
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Polycystic Liver Disease

Intervention / Treatment
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* Drug: Lanreotide Autogel 90 mg and 120 mg


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Liver volume ≥ 4 liter ≥ 20 liver cysts Symptomatic patients defined as at least 2 out of 5 of the following symptoms related to mass effect irrespective of the intensity: Abdominal distention perceived as uncomfortable Frequent abdominal pain Early satiety Nausea (with the inclusion of dyspeptic complaints) Dyspnea Diagnosed with ADPKD or ADPLD Male and female patients of 18 years and older Written informed consent Exclusion Criteria: Creatinine clearance < 20 ml/min Patient who underwent a kidney transplant and received variable doses of immunosuppressive therapy and/or present signs of rejection in the past year Hormonal replacement therapy Hormonal contraception Pregnant or lactating Presenting with an uncontrolled disease (other than ADPKD/ADPLD) Planned to undergo any surgery of the liver during study participation Planned to undergo any surgery of the KIDNEY during study participation (ADPKD patients only) Patients with known allergies to somatostatin or its analogues or any of its components Patients who received somatostatin analogues in the 6 months preceding study inclusion

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Symptomatic polycystic liver disease (PCLD) patients<br>Symptomatic polycystic liver disease (PCLD) patients | Drug: Lanreotide Autogel 90 mg and 120 mg<br>* administration of lanreotide sc every 4 weeks (28 days)<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Reduction of total liver volume after 6 months of treatment measured by means of CT-scan. | Reduction of total liver volume after 6 months measured by means of CT-scan. | 6 months | 
| Reduction of total liver volume after 12 months of treatment by means of CT-scan | Reduction of total liver volume after 12 months of treatment by means of CT-scan | 12 months | 
| Reduction of total liver volume after 18 months of treatment by means of CT-scan | Reduction of total liver volume after 18 months of treatment by means of CT-scan | 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Measurement of total liver and kidney volumes and cyst volumes at baseline. | Reduction of total liver volume Reduction of liver cyst volume Additonal reduction of total liver volume in the non responder group. Additonal reduction of liver cyst volume in the non responder group. % of patients with liver reduction > 100 ml in the non responder group. Reduction of total kidney volume (ADPKD patients only) after 6 months, 1 year and 18 months | Baseline | 
| Measurement of total liver and kidney volumes and cyst volumes after 6 months of treatment by means of CT scan | Reduction of total liver volume Reduction of liver cyst volume Additonal reduction of total liver volume in the non responder group. Additonal reduction of liver cyst volume in the non responder group. % of patients with liver reduction > 100 ml in the non responder group. Reduction of total kidney volume (ADPKD patients only) after 6 months, 1 year and 18 months | 6 months | 
| Measurement of total liver and kidney volume and cyst volume after 12 months of treatment by means of CT scan. | Reduction of total liver volume Reduction of liver cyst volume Additonal reduction of total liver volume in the non responder group. Additonal reduction of liver cyst volume in the non responder group. % of patients with liver reduction > 100 ml in the non responder group. Reduction of total kidney volume (ADPKD patients only) after 6 months, 1 year and 18 months | 12 months | 
| Measurement of total liver and kidney volumes and cyst volumes after 18 months of treatment by means of CT scan | Reduction of total liver volume Reduction of liver cyst volume Additonal reduction of total liver volume in the non responder group. Additonal reduction of liver cyst volume in the non responder group. % of patients with liver reduction > 100 ml in the non responder group. Reduction of total kidney volume (ADPKD patients only) after 6 months, 1 year and 18 months | 18 months | 
| Assessment of quality of life at baseline | Assessment of quality of life at baseline | baseline | 
| Assessment of quality of life after 6 months of treatment | Assessment of quality of life after 6 months of treatment | 6 months | 
| Assessment of quality of life after 12 months of treatment | Assessment of quality of life after 12 months of treatment | 12 months | 
| Assessment of quality of life after 18 months of treatment | Assessment of quality of life after 18 months of treatment | 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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polycystic liver disease