Somatostatin In Patients With Autosomal Dominant Polycystic Kidney Disease And Moderate To Severe Renal Insufficiency

What Was Tested

This registered phase 3 trial tested Octreotide.

It had two arms (groups): Saline solution. (placebo comparator) and Octrotide-LAR (experimental).

The registry classes the interventions as drug and other.

More on the Plan

Clinically, ADPKD is characterized by renal and extra renal manifestations.

In the kidneys, multiple cysts grow from distal and collecting tubular epithelial cells producing progressive renal enlargement with relatively initial stable renal functions.

Thereafter, both tubular and secondary interstitial damage lead to faster renal loss and end-stage renal disease (ESRD) in approximately half of all patients affected in their fifth or sixth decade of life.

Up to now, no specific therapies for ADPKD are available, but drugs like somatostatin, rapamycin, and tolvaptan targeting to growth and chloride secretion pathways are now being testing worldwide in some clinical trials.

Who Took Part

100 actual participants took part.

It was open to people of any sex aged from 18 years up to 75 years.

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

Examples of who could join: Age \> 18 years. Clinical and ultrasound diagnosis of ADPKD. Written informed consent.

The full record lists 4 inclusion and 8 exclusion criteria; among the exclusions: symptomatic urinary tract lithiasis or obstruction; current urinary tract infection; symptomatic biliary tract lithiasis.

How the Study Was Run

It was run as a randomized, parallel-group and quadruple-blind study.

Random assignment meant chance, not the researchers, decided who got Octreotide and who got the comparison, which limits bias.

The Octreotide group and the comparison group ran side by side rather than switching.

Blinding on both sides kept the participants and researchers unaware of who received Octreotide.

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

It started in 2011-05 with completion listed for 2017-10-18.

It ran at 6 sites in Italy (6).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Total kidney volume (TKV) change (delta TKV) as assessed by spiral computed tomography (spiral CT) scan (time frame: At 0, 12 and 36 month). Rate of GFR decline as assessed by serial measurements of the iohexol plasma clearance (time frame: At 0, 12, 24 and 36 month).

It also tracked 4 secondary outcomes, for example Total renal cyst volume, Total renal parenchyma volume, Total renal intermediate volume and Total liver and liver cyst volumes.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Octreotide in 100 people.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

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