NCT00426153

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
42

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jan 2007

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2007

Completed
21 days until next milestone

First Submitted

Initial submission to the registry

January 22, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 24, 2007

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2008

Completed
4.1 years until next milestone

Results Posted

Study results publicly available

November 21, 2012

Completed
Last Updated

November 21, 2012

Status Verified

October 1, 2012

Enrollment Period

1.8 years

First QC Date

January 22, 2007

Results QC Date

October 22, 2012

Last Update Submit

October 22, 2012

Conditions

Polycystic Kidney, Autosomal DominantPolycystic Liver DiseaseHepatomegalyLiver DiseasesKidney, PolycysticAbdominal Pain

Outcome Measures

Primary Outcomes (1)

  • Percent Change in Liver Volume

    Percent change from baseline in liver volume, measured in milliliters by Magnetic Resonance Imaging (MRI)or Computed Tomography (CT) scans

    Baseline, 12 months

Secondary Outcomes (3)

  • Percent Change in Renal Volume

    Baseline, 12 months

  • Percent Change in Glomerular Filtration Rate (GFR)

    Baseline, 12 months

  • Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores

    Baseline, 12 months

Study Arms (2)

Octreotide

ACTIVE COMPARATOR

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

Drug: Octreotide

Placebo

PLACEBO COMPARATOR

Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year

Drug: Placebo

Interventions

OctreotideDRUG

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

Also known as: Octreotide LAR® Depot
Octreotide
PlaceboDRUG

Participants received an injection of placebo (sham) medication intramuscularly every 28 days (+/- 5 day) for one year

Also known as: Placebo injection
Placebo

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related Publications (3)

  • Hogan MC, Masyuk TV, Page L, Holmes DR 3rd, Li X, Bergstralh EJ, Irazabal MV, Kim B, King BF, Glockner JF, Larusso NF, Torres VE. Somatostatin analog therapy for severe polycystic liver disease: results after 2 years. Nephrol Dial Transplant. 2012 Sep;27(9):3532-9. doi: 10.1093/ndt/gfs152. Epub 2012 Jul 6.

    PMID: 22773240RESULT

  • 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: 39356039DERIVED

  • Hogan MC, Masyuk T, Bergstralh E, Li B, Kremers WK, Vaughan LE, Ihrke A, Severson AL, Irazabal MV, Glockner J, LaRusso NF, Torres VE. Efficacy of 4 Years of Octreotide Long-Acting Release Therapy in Patients With Severe Polycystic Liver Disease. Mayo Clin Proc. 2015 Aug;90(8):1030-7. doi: 10.1016/j.mayocp.2015.05.011. Epub 2015 Jul 9.

    PMID: 26166166DERIVED

MeSH Terms

Conditions

Polycystic Kidney, Autosomal DominantPolycystic liver diseaseHepatomegalyLiver DiseasesPolycystic Kidney DiseasesAbdominal Pain

Interventions

Octreotide

Condition Hierarchy (Ancestors)

Kidney Diseases, CysticKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCiliopathiesGenetic Diseases, InbornDigestive System DiseasesHypertrophyPathological Conditions, AnatomicalPathological Conditions, Signs and SymptomsPainNeurologic ManifestationsSigns and SymptomsSigns and Symptoms, Digestive

Intervention Hierarchy (Ancestors)

Peptides, CyclicMacrocyclic CompoundsPolycyclic CompoundsPeptidesAmino Acids, Peptides, and Proteins

Results Point of Contact

Title
Marie Hogan, MD, PhD, Assistant Prof of Medicine, College of Medicine
Organization
Mayo Clinic
hogan.marie@mayo.edu
507-266-9364

Study Officials

  • Marie C. Hogan, M.D., Ph.D.

    Mayo Clinic

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, PhD, Assistant Professor of Medicine, College of Medicine

Study Record Dates

First Submitted

January 22, 2007

First Posted

January 24, 2007

Study Start

January 1, 2007

Primary Completion

October 1, 2008

Study Completion

October 1, 2008

Last Updated

November 21, 2012

Results First Posted

November 21, 2012

Record last verified: 2012-10

Locations

US(1)