Study of Lanreotide to Treat Polycystic Kidney Disease

What Was Tested

This registered phase 3 trial tested Lanreotide.

Autosomal dominant polycystic kidney disease (ADPKD) is characterized by progressive cyst formation in both kidneys, in most patients leading to end stage renal disease.

It is the most common hereditary renal disease with a prevalence of approximately 1 in 1,000 persons.

The majority of patients also have progressive cyst formation in the liver, leading to pain, gastrointestinal discomfort and sometimes the need for liver transplantation.

It had two arms (groups): standard care (no intervention) and Lanreotide (experimental).

Subjects in this arm will receive standard care.

The registry classes the interventions as drug.

More on the Plan

Aims: First, to determine whether Lanreotide attenuates progression of the renal phenotype in ADPKD patients as measured by change in rate of renal function decline and change in renal volume growth.

Second, to determine whether Lanreotide modifies progression of the liver phenotype in the subset of ADPKD patients with moderate to severe polycystic liver disease as measured by change in liver volume.

Intervention: Patients will be randomized (1:1) into two groups.

The other group of patients will receive standard care.

Who Took Part

300 planned participants were the target.

It was open to people of any sex aged from 18 years up to 60 years.

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

Examples of who could join: Diagnosis of ADPKD, based upon the modified Ravine criteria. Age between 18 and 60 years. Providing informed consent.

The full record lists 4 inclusion and 15 exclusion criteria; among the exclusions: patients who, in the opinion of the study investigator may present a safety risk; patients taking other experimental (i.e.,non approved by fda/ema or indication of adpkd) therapies; patients having used lanreotide (or another somatostatin analogue) in the 3 months before study start.

How the Study Was Run

The set-up was randomized, parallel-group and open-label.

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

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

Nothing was hidden in this open-label Lanreotide study: the participants and staff both knew the treatment.

It started in 2012-06 with completion listed for 2017-08.

It ran at 4 sites in Netherlands (4).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Change in renal function (time frame: serial eGFR measurements from month 3 until end of treatment visit (month 30)).

It also tracked 6 secondary outcomes, for example change in renal volume, change in liver volume, change in quality of life, tolerance, change in renal function and Incidence of worsening renal function.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Lanreotide in 300 people.

Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.

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