Octreotide in Severe Polycystic Liver Disease

What Was Tested

In this phase 2, phase 3 trial, researchers set out to test Octreotide.

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.

It had two arms (groups): Octreotide (active comparator) and Placebo (placebo comparator).

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

The registry classes the interventions as drug.

More on the Plan

(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.

Who Took Part

The registry lists 42 actual participants took part.

It was open to people of any sex aged from 18 years up to 80 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Age - 18 years and older. Not a candidate for or declining surgical intervention.

The full record lists 4 inclusion and 10 exclusion criteria; among the exclusions: women of childbearing potential or their sexual partners who are unwilling to employ adequate contraception; symptomatic gallstones or biliary sludge; variceal bleeding or hepatic encephalopathy within prior 30 days.

How the Study Was Run

Design: randomized, parallel-group and triple-blind.

Because people in this Octreotide study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Groups ran in parallel: each of the participants stayed in one arm throughout.

As a double-blind Octreotide study, the assignments were hidden from both participants and investigators until the end.

Those kept unaware of assignments: participant, care provider and investigator.

It started in 2007-01 with completion listed for 2008-10.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Percent Change in Liver Volume (time frame: Baseline, 12 months).

It also tracked 3 secondary outcomes, for example Percent Change in Renal Volume, Percent Change in Glomerular Filtration Rate (GFR) and Change in Subject Reported Outcomes Using Mean Health Related Quality of Life (HRQoL) Scores.

What This Means in Plain Language

Put simply, this is a phase 2, phase 3, randomized trial of Octreotide in 42 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

More context: the Octreotide compound page, all human trials.