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Everolimus Added to Long Acting Octreotide as a Volume Reducing Treatment of Polycystic Livers

Phase 2
18 Years
70 Years
Open (Enrolling)
Polycystic Liver Disease

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Trial Information

Everolimus Added to Long Acting Octreotide as a Volume Reducing Treatment of Polycystic Livers

This is a single center randomized, open-label, parallel study comparing the safety and
efficacy of everolimus-octreotide LAR treatment to monotherapy octreotide LAR in adult
symptomatic patients with polycystic livers because of polycystic liver disease (PCLD).

We aim to include 44 patients affected by a polycystic liver either due to PCLD, 22
patients in the combination group and 22 patients in the mono therapy group.The duration of
the trial will be 52 weeks. The treatment will be 48 weeks and the last control visit will
take place four weeks after the treatment.

Inclusion Criteria:

- 18 < 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

Exclusion Criteria:

- 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

- 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

- 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 <

- 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

- Co-medication with strong inhibitor of CYP3A4 and or P-gp like voriconazole,
ketoconazole, diltiazem, verapamil, erythromycin or with a strong CYP3A4 and or P-gp
inductor like rifampicin

- Known hypersensitivity to everolimus or one of its excipients

- Enrolment in another clinical trial of an investigational agent while participating
in this study

- Moderate or severe reaction on contrast in medical history

- Treatment with I131 during the course of the trial

- Use of metformin

- Morbus Kahler or Morbus Waldenstrom with excretion of light chains in urine in
medical history

- Kidney dysfunction (MDRD-GFR < 60 ml/min/1.73m2 and ECC < 60 ml/min, calculated by
the Cockcroft-Gault formula); in case of decreased body muscle mass, exact ECC is
measured using serum and urine creatinine

Type of Study:


Study Design:

Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Liver volume

Outcome Description:

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

Outcome Time Frame:

at baseline and at 12 months

Safety Issue:


Principal Investigator

Joost PH Drenth, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Radboud University


Netherlands: Medical Ethics Review Committee (METC)

Study ID:




Start Date:

June 2010

Completion Date:

June 2012

Related Keywords:

  • Polycystic Liver Disease
  • liver cyst
  • Liver Diseases
  • Cysts