MedDataX Logo

Octreotide in Severe Polycystic Liver Disease

NCT ID: NCT00426153

Last Updated: 2012-11-21

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

42 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-01-31

Study Completion Date

2008-10-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Polycystic Kidney, Autosomal Dominant Polycystic Liver Disease Hepatomegaly Liver Diseases Kidney, Polycystic Abdominal Pain

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Octreotide

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

Group Type ACTIVE_COMPARATOR

Octreotide

Intervention Type DRUG

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

Placebo

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

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Octreotide

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

Intervention Type DRUG

Placebo

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

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Octreotide LAR® Depot Placebo injection

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Novartis

INDUSTRY

Sponsor Role collaborator

National Center for Research Resources (NCRR)

NIH

Sponsor Role collaborator

Mayo Clinic

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Marie Hogan

MD, PhD, Assistant Professor of Medicine, College of Medicine

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

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

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type RESULT
PMID: 22773240 (View on PubMed)

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.

Reference Type DERIVED
PMID: 39356039 (View on PubMed)

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.

Reference Type DERIVED
PMID: 26166166 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

UL1RR024150

Identifier Type: NIH

Identifier Source: secondary_id

View Link

06-004128

Identifier Type: -

Identifier Source: org_study_id