Everolimus and LongActing Octreotide Trial in Polycystic Livers

NCT01157858 | Completed Phase 2 DMC

• 1 other identifier: CSMS995 ANLIIT
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this study is to reduce polycystic liver volume by treating with octreotide, whether or not combined with everolimus; to assess whether combination therapy of everolimus and octreotide gives a bigger reduction of polycystic liver volume than octreotide monotherapy.

Conditions

Polycystic Liver Disease

Keywords

liver cyst

Outcome Measures

Primary Outcomes (1)

• Liver volume

  change of total liver volume in terms of percentage from baseline to 12 months as determined by CT

  at baseline and at 12 months

Secondary Outcomes (4)

• Symptoms

  baseline and 12 months

• Quality of Life

  baseline and 12 months

• Responders

  baseline and 12 months

• Adverse events

  During 12 months of treatment

Study Arms (2)

Everolimus + octreotide LAR

ACTIVE COMPARATOR

Octreotide LAR combined with everolimus

Drug: Everolimus; Drug: Octreotide LAR

Octreotide LAR

ACTIVE COMPARATOR

Octreotide LAR monotherapy

Drug: Octreotide LAR

Interventions

Everolimus DRUG

2.5 mg every day orally

Also known as: Afinitor

Everolimus + octreotide LAR

Octreotide LAR DRUG

40 mg every 28 days IM

Also known as: Sandostatine LAR

Everolimus + octreotide LAR; Octreotide LAR

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \< age ≤ 70 years
• Polycystic liver disease (PCLD), defined as ≥ 20 liver cysts
• Total liver volume must be at least 2500 mL
• Symptomatic defined as ECOG-PS ≥ 1 (see fig 3.1)38, and having at least three out of ten PCLD symptoms:
• Abdominal pain
• Abdominal distension
• Abdominal fullness
• Dyspnea
• Early satiety
• Back pain
• Nausea/vomiting
• Anorexia
• Weight loss
• Jaundice
• Informed consent, patients are willing and able to comply with the study drug regimen and all other study requirements

You may not qualify if:

• ADPKD patients
• Use of oral anticonceptives or estrogen supplementation
• Females who are pregnant or breast-feeding or patients of reproductive potential not employing an effective method of birth control. Women of childbearing potential must have a negative serum pregnancy test within 48 hours prior to the administration of study medication.
• Intervention (aspiration or surgical intervention) within three months before baseline
• Treatment with somatostatin analogues within three months before baseline
• Patients with a kidney transplant
• History or other evidence of chronic pulmonary disease associated with functional limitation
• History of severe cardiac disease (eg, NYHA Functional Class III or IV, myocardial infarction within 6 months, ventricular tachyarrhythmias requiring ongoing treatment, unstable angina or other significant cardiovascular diseases). In addition, patients with documented or presumed coronary artery disease or cerebrovascular disease should not be enrolled.
• History or other evidence of severe illness or any other conditions which would make the patient, in the opinion of the investigator, unsuitable for the study
• Symptomatic gallstones (octreotide decreases gall bladder volume)
• Hypercholesterolemia (fasting cholesterol \> 8 mmol/l) or hypertriglyceridaemia (\> 5 mmol/l) not controlled by lipid lowering therapy
• Granulocytopenia (white blood cell \< 3,000/mm3) or thrombocytopenia (platelets \< 100,000/mm3)
• Infection with hepatitis B, hepatitis C, HIV, TBC (in medical history)
• Mental illness that interferes with the patient ability to comply with the protocol
• Drug or alcohol abuse within one year of baseline

Sponsors & Collaborators

• Radboud University Medical Center: lead
• Novartis: collaborator

Study Sites (1)

Department of Gastroenterology and Hepatology, Radboud University Nijmegen Medical Centre

Nijmegen, 6500 HB, Netherlands

Location

Related Publications (2)

• St Pierre K, Cashmore BA, Bolignano D, Zoccali C, Ruospo M, Craig JC, Strippoli GF, Mallett AJ, Green SC, Tunnicliffe DJ. Interventions for preventing the progression of autosomal dominant polycystic kidney disease. Cochrane Database Syst Rev. 2024 Oct 2;10(10):CD010294. doi: 10.1002/14651858.CD010294.pub3.

  PMID: 39356039 DERIVED

• Chrispijn M, Drenth JP. Everolimus and long acting octreotide as a volume reducing treatment of polycystic livers (ELATE): study protocol for a randomized controlled trial. Trials. 2011 Nov 21;12:246. doi: 10.1186/1745-6215-12-246.

  PMID: 22104015 DERIVED

MeSH Terms

Conditions

Polycystic liver disease

Interventions

Everolimus

Intervention Hierarchy (Ancestors)

Sirolimus; Macrolides; Lactones; Organic Chemicals

Study Officials

• Joost PH Drenth, MD, PhD

  Radboud University Medical Center

  PRINCIPAL INVESTIGATOR

• Melissa Chrispijn, MD

  Radboud University Medical Center

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD

Study Record Dates

• First Submitted: July 6, 2010
• First Posted: July 7, 2010
• Study Start: June 1, 2010
• Primary Completion: July 1, 2012
• Study Completion: July 1, 2012
• Last Updated: June 29, 2015

Record last verified: 2015-06

Locations

NL (1)