Tolvaptan-Octreotide LAR Combination in ADPKD

What Was Tested

This registered phase 2 trial tested Octreotide.

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is a leading cause of End Stage Kidney Disease (ESKD) worldwide.

Elevated levels of 3', 5' - cyclic AMP (cAMP) play a central role in the pathogenesis and progression of the disease.

It had two arms (groups): Tolvaptan plus Octreotide LAR / Tolvaptan plus Placebo (experimental) and Tolvaptan plus placebo/Tolvaptan plus Octreotide LAR (experimental).

The registry classes the interventions as drug and other.

Who Took Part

Enrollment: 20 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

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

Examples of who could join: Adult (\>18-yr-old) men and women, with a clinical and ultrasonographic diagnosis of ADPKD;. Written informed consent.

The full record lists 7 inclusion and 16 exclusion criteria; among the exclusions: patients with concomitant systemic, renal parenchymal or urinary tract disease;; hemorrhagic or complicated cysts which might acutely affect kidney function and volumes;; QT-related ECG abnormalities;.

How the Study Was Run

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

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

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

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, investigator and outcomes assessor.

It started in 2018-12-12 with completion listed for 2021-12-23.

It ran at 1 site in Italy.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Glomerular Filtration Rate (GFR) (time frame: Changes from 4 weeks before randomization at baseline, 1,4,8,9,12 and 16 weeks after the randomization).

It also tracked 1 secondary outcome, for example Total Kidney Volume (TKV).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Octreotide in 20 people.

Its size is small, so on its own it can only give an early look.

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.