Clinical trial NCT01315795: Lanreotide Autogel in the Treatment of Symptomatic Polycystic Liver Disease
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Polycystic Liver Disease
Keywords: polycystic liver disease
Lead sponsor: Universitaire Ziekenhuizen KU Leuven
Sponsor class: OTHER
Collaborators: name: Ipsen
First posted: 2011-03-15
Enrollment: 59
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Reduction of total liver volume after 6 months of treatment measured by means of CT-scan.
timeFrame: 6 months
measure: Reduction of total liver volume after 12 months of treatment by means of CT-scan
timeFrame: 12 months
measure: Reduction of total liver volume after 18 months of treatment by means of CT-scan
timeFrame: 18 months
Secondary outcomes:
measure: Measurement of total liver and kidney volumes and cyst volumes at baseline.
timeFrame: Baseline
measure: Measurement of total liver and kidney volumes and cyst volumes after 6 months of treatment by means of CT scan
timeFrame: 6 months
measure: Measurement of total liver and kidney volume and cyst volume after 12 months of treatment by means of CT scan.
timeFrame: 12 months
measure: Measurement of total liver and kidney volumes and cyst volumes after 18 months of treatment by means of CT scan
timeFrame: 18 months
measure: Assessment of quality of life at baseline
timeFrame: baseline
measure: Assessment of quality of life after 6 months of treatment
timeFrame: 6 months
measure: Assessment of quality of life after 12 months of treatment
timeFrame: 12 months
measure: Assessment of quality of life after 18 months of treatment
timeFrame: 18 months
Other outcomes:
Locations:
city: Leuven
country: Belgium
facility: UZ Leuven, Gasthuisberg
state: Provincie Vlaams-Brabant
Study officials:
affiliation: UZ Leuven, Gasthuisberg
name: Frederik Nevens, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: van Keimpema L, Nevens F, Vanslembrouck R, van Oijen MG, Hoffmann AL, Dekker HM, de Man RA, Drenth JP. Lanreotide reduces the volume of polycystic liver: a randomized, double-blind, placebo-controlled trial. Gastroenterology. 2009 Nov;137(5):1661-8.e1-2. doi: 10.1053/j.gastro.2009.07.052. Epub 2009 Jul 29.
citation: Temmerman F, Ho TA, Vanslembrouck R, Coudyzer W, Billen J, Dobbels F, van Pelt J, Bammens B, Pirson Y, Nevens F. Lanreotide Reduces Liver Volume, But Might Not Improve Muscle Wasting or Weight Loss, in Patients With Symptomatic Polycystic Liver Disease. Clin Gastroenterol Hepatol. 2015 Dec;13(13):2353-9.e1. doi: 10.1016/j.cgh.2015.05.039. Epub 2015 Jun 12.