A Trial to Assess the Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With PLD

What Was Tested

This registered phase 2, phase 3 trial tested Octreotide.

In the Treatment Period of the trial, participants will be allocated at random to 1 of the 3 treatment arms in a 1:1:1 ratio.

After completing the Treatment Period (53 weeks) participants may proceed to a 120-week open-label extension part of the trial and then only receive the same CAM2029 treatment.

It had three arms (groups): CAM2029 once weekly (experimental), CAM2029 once every 2 weeks (experimental) and Placebo (placebo comparator).

The registry classes the interventions as drug.

Who Took Part

The registry lists 71 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: Male or female patient, ≥18 years at screening. Not a candidate for, or not willing to undergo, surgical intervention for hepatic cysts during the trial.

The full record lists 4 inclusion and 8 exclusion criteria; among the exclusions: surgical intervention for pld within 3 months before screening; treatment with a somatostatin analogue (ssa) within 3 months before screening; non-responsive to previous treatment of pld with an ssa as per the investigator's assessment.

How the Study Was Run

Design: randomized, parallel-group and quadruple-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.

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

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 2022-06-28 with completion listed for 2025-02-05.

It ran at 11 sites in United States (6), Germany (3), Belgium (1) and Netherlands (1).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Height-adjusted Total Liver Volume (htTLV) (time frame: From screening until treatment week 53).

It also tracked 13 secondary outcomes, for example PLD Symptom (PLD-S) Score, htTLV, PLD-S, Height-adjusted Total Kidney Volume (htTKV), Total Liver Cyst Volume and Estimated Glomerular Filtration Rate (eGFR).

What This Means in Plain Language

Put simply, this is a phase 2, phase 3, randomized trial of Octreotide in 71 people.

Its status is active not recruiting; results are posted on the registry, and this page does not restate them.

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