Polycystic Liver Disease
Conditions
Keywords
polycystic liver disease
Brief summary
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.
Interventions
DRUGLanreotide Autogel 90 mg and 120 mg
administration of lanreotide sc every 4 weeks (28 days)
Sponsors
Ipsen
Universitaire Ziekenhuizen KU Leuven
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Reduction of total liver volume after 6 months of treatment measured by means of CT-scan. | 6 months | Reduction of total liver volume after 6 months measured 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 12 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 | Reduction of total liver volume after 18 months of treatment by means of CT-scan |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Measurement of total liver and kidney volumes and cyst volumes after 18 months of treatment by means of CT scan | 18 months | 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 |
| Assessment of quality of life at baseline | baseline | Assessment of quality of life at baseline |
| Measurement of total liver and kidney volumes and cyst volumes at baseline. | 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 |
| Assessment of quality of life after 12 months of treatment | 12 months | Assessment of quality of life after 12 months of treatment |
| Assessment of quality of life after 18 months of treatment | 18 months | Assessment of quality of life after 18 months of treatment |
| Assessment of quality of life after 6 months of treatment | 6 months | Assessment of quality of life after 6 months of treatment |
| Measurement of total liver and kidney volumes and cyst volumes after 6 months of treatment by means of CT scan | 6 months | 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 |
| Measurement of total liver and kidney volume and cyst volume after 12 months of treatment by means of CT scan. | 12 months | 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 |
Countries
Belgium
Outcome results
None listed