Octreotide in Severe Polycystic Liver Disease

Brief Title

Octreotide in Severe Polycystic Liver Disease

Official Title

Pilot Study Of Long-Acting Octreotide (Octreotide LAR® Depot) In The Treatment Of Patients With Severe Polycystic Liver Disease

Brief Summary

      This study will evaluate the effect of Octreotide LAR® on the liver volumes of patients with
      severe polycystic liver disease who are not candidates or decline surgical treatments such as
      liver cyst fenestration, liver resection or liver transplantation. A total of 42 patients
      will be recruited -14 who will receive placebo and 28 the study drug. Preliminary evidence
      indicates that this drug is safe and non-toxic in other disease states. Treatment with this
      drug holds promise not only for individuals with liver involvement, but also for many more
      patients with polycystic kidney disease.
    

Detailed Description

      The primary aim of this study is to compare the effect of Octreotide LAR® Depot on the liver
      volume of patients with severe polycystic liver disease who are not candidates or decline
      surgical treatments such as liver cyst fenestration, liver resection or liver transplantation
      compared with placebo. The secondary aims of the study are: (1)Assess the effect of
      Octreotide LAR® Depot on the total kidney volume and iothalamate clearance in patients with
      polycystic kidney disease associated with severe polycystic liver disease who are not
      candidates or decline surgical treatments such as liver cyst fenestration, liver resection or
      liver transplantation. (2)Evaluate quality of life changes associated with the administration
      of Octreotide LAR® Depot in these patients. (3)Assess toxicity of Octreotide LAR® Depot in
      patients with polycystic liver disease (PLD).

      Note: Subjects who completed this 1 year randomized trial were offered enrollment into an
      open-label (all subjects received Octreotide) extension trial for an additional two years of
      treatment.
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

Percent Change in Liver Volume

Secondary Outcome

 Percent Change in Renal Volume

Condition

Polycystic Kidney, Autosomal Dominant

Intervention

Octreotide

Study Arms / Comparison Groups

 Octreotide
Description:  Participants received Octreotide LAR® Depot injections (up to 40 mg) intramuscularly every 28 days (+/- 5 days) for one year

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

42

Start Date

January 2007

Completion Date

October 2008

Primary Completion Date

October 2008

Eligibility Criteria

        Inclusion Criteria:

          -  Age - 18 years and older

          -  Diagnosis of Polycystic Liver Disease (PLD) associated with ADPKD or isolated
             Autosomal Dominant Polycystic liver Disease (ADPLD)

          -  Severe PLD defined as a liver volume greater than 4000 mL or symptomatic disease due
             to mass effects from hepatic cysts

          -  Not a candidate for or declining surgical intervention

        Exclusion Criteria:

          -  Women of childbearing potential or their sexual partners who are unwilling to employ
             adequate contraception

          -  Creatinine greater than 3mg/dL or hemodialysis dependent

          -  Cancer or major systemic disease that could prevent completion of the planned
             follow-up or interfere with data collection or interpretation

          -  Uncontrolled diabetes mellitus as defined by blood glucose levels of greater than or
             equal to 250 mg/dL on 2 or more consecutive daily readings despite antidiabetic
             therapy

          -  Neurologic/psychologic conditions preventing appropriate informed consent

          -  Symptomatic gallstones or biliary sludge

          -  Variceal bleeding or hepatic encephalopathy within prior 30 days

          -  Uncontrolled hypertension (Systolic blood pressure greater than 160 mmHg; Diastolic
             blood pressure greater than 100 mmHg)

          -  Current, or prior use of somatostatin analogue (octreotide, lanreotide) in past 6
             months

          -  History of significant adverse reaction to a somatostatin analogue
      

Gender

All

Ages

18 Years - 80 Years

Accepts Healthy Volunteers

No

Contacts

Marie C. Hogan, M.D., Ph.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00426153

Organization ID

06-004128

Secondary IDs

UL1RR024150

Responsible Party

Principal Investigator

Study Sponsor

Mayo Clinic

Collaborators

 Novartis

Study Sponsor

Marie C. Hogan, M.D., Ph.D., Principal Investigator, Mayo Clinic


Verification Date

October 2012