Pasireotide LAR in Severe Polycystic Liver Disease

What Was Tested

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

The purpose of this study is to compare SOM230 treatment to placebo.

It had two arms (groups): Pasireotide LAR (SOM230) (active comparator) and placebo injection (placebo comparator).

Active Pasireotide LAR.

The registry classes the interventions as drug.

More on the Plan

Pasireotide (SOM230) is a novel multi-receptor-targeted analog that has high affinity for four of the five SST receptor subtypes (SSTr1, SSTr2, SSTr3 and SSTr5); it has a 40-fold higher affinity and 158-fold higher functional activity for the SST5 receptor than octreotide.

Because of its broad receptor binding profile, pasireotide may be more potent in Polycystic Liver Disease (PLD) than octreotide.

In this randomized double blind placebo controlled trial the investigators will compare SOM230 treatment to placebo for 12 months in patients with PLD.

The primary endpoints will be assessed at 12 months and patients receiving placebo then crossed over to SOM230, permitting all participants to receive SOM230 for the subsequent two years.

Who Took Part

The registry lists 48 actual participants took part.

It was open to people of any sex aged from 18 years up to 100 years.

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

Examples of who could join: Male or female Age ≥ 18 years. Not a candidate for or declining surgical intervention. Capable of providing informed consent. Life expectancy ≥ 12 weeks. Adequate end organ function as defined by:.

The full record lists 22 inclusion and 36 exclusion criteria; among the exclusions: patients will be considered ineligible for this study if they meet any of the following criteria:; patients with a known hypersensitivity to sst analogs or any component of the pasireotide lar or sq formulations; patients with known malabsorption syndrome, short bowel or chologenic diarrhea not controlled by specific therapeutic means.

How the Study Was Run

The set-up was randomized, crossover and double-blind.

The participants were allocated by chance, so the Pasireotide group and the comparison group should start out alike.

In the crossover set-up each of the participants took both Pasireotide and the comparison in turn, acting as their own control.

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

Those kept unaware of assignments: participant and investigator.

It started in 2012-08 with completion listed for 2018-09.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Change in Liver Volume (time frame: baseline , 12 month). Change in Kidney Volume (time frame: baseline to 12 months).

It also tracked 6 secondary outcomes, for example Percentage Change in Estimated Glomerular Filtration Rate (eGFR), Percentage Change in Serum Creatinine, Percent Change in Blood Glucose, Percentage Change in Hemoglobin A1C, Percentage Change in Heart Rate and Change in Quality of Life.

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Pasireotide in 48 people.

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

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