title: Rationale and design of the RESOLVE trial: lanreotide as a volume reducing treatment for polycystic livers in patients with autosomal dominant polycystic kidney disease.
abstract: BACKGROUND
  A large proportion of patients with autosomal dominant polycystic kidney disease (ADPKD) suffers from polycystic liver disease. Symptoms arise when liver volume increases. The somatostatin analogue lanreotide has proven to reduce liver volume in patients with polycystic liver disease. However, this study also included patients with isolated polycystic liver disease (PCLD). The RESOLVE trial aims to assess the efficacy of lanreotide treatment in ADPKD patients with symptomatic polycystic livers. In this study we present the design of the RESOLVE trial.
  
  
  METHODS
  This open-label clinical trial evaluates the effect of 6 months of lanreotide in ADPKD patients with symptomatic polycystic livers. Primary outcome is change in liver volume determined by computerised tomography-volumetry. Secondary outcomes are changes in total kidney volume, kidney intermediate volume and renal function. Furthermore, urinary (NGAL, α1-microglobulin, KIM-1, H-FABP, MCP-1) and serum (fibroblast growth factor 23) biomarkers associated with ADPKD disease severity are assessed to investigate whether these biomarkers predict treatment responses to lanreotide. Moreover, safety and tolerability of the drug in ADPKD patients will be assessed.
  
  
  CONCLUSIONS
  We anticipate that lanreotide is an effective therapeutic option for ADPKD patients with symptomatic polycystic livers and that this trial aids in the identification of patient related factors that predict treatment response.
  
  
  BACKGROUND
  Clinical trials.gov NCT01354405.
authors.affiliation: Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Center, Nijmegen, the Netherlands.
authors.forename: Tom J G
authors.initials: TJ
authors.lastname: Gevers
authors.forename: Melissa
authors.initials: M
authors.lastname: Chrispijn
authors.forename: Jack F M
authors.initials: JF
authors.lastname: Wetzels
authors.forename: Joost P H
authors.initials: JP
authors.lastname: Drenth
mesh_terms: D000209:Acute-Phase Proteins / Q000652:urine; D000293:Adolescent; D000328:Adult; D000368:Aged; D000510:Alpha-Globulins / Q000652:urine; D000970:Antineoplastic Agents / Q000494:pharmacology / Q000627:therapeutic use*; D015415:Biomarkers / Q000097:blood / Q000652:urine; D018932:Chemokine CCL2 / Q000652:urine; D003560:Cysts / Q000150:complications / Q000188:drug therapy* / Q000378:metabolism / Q000473:pathology*; D000074026:Fatty Acid Binding Protein 3; D050556:Fatty Acid-Binding Proteins / Q000652:urine; D000089703:Fibroblast Growth Factor-23; D005346:Fibroblast Growth Factors / Q000097:blood; D005919:Glomerular Filtration Rate; D000072596:Hepatitis A Virus Cellular Receptor 1; D006801:Humans; D007668:Kidney / Q000473:pathology; D016014:Linear Models; D000071068:Lipocalin-2; D054834:Lipocalins / Q000652:urine; D008099:Liver / Q000473:pathology*; D008107:Liver Diseases / Q000150:complications / Q000188:drug therapy* / Q000378:metabolism / Q000473:pathology*; D008562:Membrane Glycoproteins / Q000652:urine; D008875:Middle Aged; D009929:Organ Size / Q000187:drug effects; D010456:Peptides, Cyclic / Q000494:pharmacology / Q000627:therapeutic use*; D016891:Polycystic Kidney, Autosomal Dominant / Q000150:complications / Q000378:metabolism / Q000473:pathology*; D011518:Proto-Oncogene Proteins / Q000652:urine; D011991:Receptors, Virus; D012107:Research Design; D012720:Severity of Illness Index; D013004:Somatostatin / Q000031:analogs & derivatives* / Q000494:pharmacology / Q000627:therapeutic use; D018709:Statistics, Nonparametric; D055815:Young Adult
chemical_list: D000209:Acute-Phase Proteins; D000510:Alpha-Globulins; D000970:Antineoplastic Agents; D015415:Biomarkers; D018932:Chemokine CCL2; C493408:FABP3 protein, human; D000074026:Fatty Acid Binding Protein 3; D050556:Fatty Acid-Binding Proteins; C491143:HAVCR1 protein, human; D000072596:Hepatitis A Virus Cellular Receptor 1; C489506:LCN2 protein, human; D000071068:Lipocalin-2; D054834:Lipocalins; D008562:Membrane Glycoproteins; D010456:Peptides, Cyclic; D011518:Proto-Oncogene Proteins; D011991:Receptors, Virus; C016001:alpha-1-microglobulin; C060347:lanreotide; D013004:Somatostatin; D005346:Fibroblast Growth Factors; D000089703:Fibroblast Growth Factor-23