Lanreotide Autogel in the Treatment of Symptomatic Polycystic Liver Disease (LOCKCYST)

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

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 Overview

• Status: Completed
• Conditions: Polycystic Liver Disease
• Intervention / Treatment: Drug: Lanreotide Autogel 90 mg and 120 mg

• Study Type: Interventional
• Enrollment (Actual): 59
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Belgium
  • Provincie Vlaams-Brabant
    • Leuven, Provincie Vlaams-Brabant, Belgium, 3000
      • UZ Leuven, Gasthuisberg

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Universitaire Ziekenhuizen KU Leuven
• Collaborators: Ipsen
• Investigators: Principal Investigator: Frederik Nevens, MD, PhD, UZ Leuven, Gasthuisberg

Publications and helpful links

General Publications

• 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.
• 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.

Helpful Links

• Lanreotide reduces the volume of polycystic liver: a randomized, double-blind, placebo-controlled trial.

Study record dates

Study Major Dates

• Study Start: March 1, 2011
• Primary Completion (Actual): July 1, 2014
• Study Completion (Actual): July 1, 2014

Study Registration Dates

• First Submitted: January 11, 2011
• First Submitted That Met QC Criteria: March 14, 2011
• First Posted (Estimate): March 15, 2011

Study Record Updates

• Last Update Posted (Estimate): July 8, 2014
• Last Update Submitted That Met QC Criteria: July 7, 2014
• Last Verified: July 1, 2014

More Information

Terms related to this study

• Keywords: polycystic liver disease
• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms; Pathological Conditions, Anatomical; Liver Diseases; Cysts; Antineoplastic Agents; Lanreotide
• Other Study ID Numbers: 2010-024604-10