Efficacy and Safety of Pasireotide Administered Monthly in Patients With Cushing's Disease

What Was Tested

In this phase 3 trial, researchers set out to test Pasireotide.

It had two arms (groups): one (experimental) and one (experimental).

The registry classes the interventions as drug.

Who Took Part

Enrollment: 150 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: Karnofsky performance status ≥ 60 (i.e. requires occasional assistance, but is able to care for most of their personal needs). For patients on medical treatment for Cushing's disease the following washout periods must be completed before screening assessments are performed. Inhibitors of steroidogenesis (ketoconazole, metyrapone): 1 week. Pituitary directed agents: Dopamine agonists (bromocriptine, cabergoline) and PPARγ agonists (rosiglitazone or pioglitazone): 4 weeks. Octreotide LAR, Lanreotide SR and Lanreotide autogel: 14 weeks.

The full record lists 6 inclusion and 7 exclusion criteria; among the exclusions: patients who are considered candidates for surgical treatment at the time of study entry; patients who have received pituitary irradiation within the last ten years prior to visit 1; patients who have had any previous pasireotide treatment.

How the Study Was Run

The set-up was randomized, parallel-group and triple-blind.

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

Groups ran in parallel: each of the participants stayed in one arm throughout.

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

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

It started in 2011-11-04 with completion listed for 2016-12-21.

It ran at 82 sites in United States (12), Japan (12), France (6), Belgium (5), Italy (5) and Brazil (4).

Its stated primary purpose is treatment.

What Was Measured

It also tracked 28 secondary outcomes, for example Actual Change in Mean Urinary Free Cortisol (mUFC) From Baseline, Percentage Change in Mean Urinary Free Cortisol (mUFC) From Baseline, Percentage of Patients Who Attain mUFC ≤ 1.0 x ULN, Percent of Participants Attaining a Duration of Controlled or Partially Controlled Response at Indicated Time Points, Percentage Change From Baseline on Plasma Adrenocorticotropic Hormone (ACTH) Over Time and Percentage Change From Baseline on Serum Cortisol Over Time.

What This Means in Plain Language

Put simply, this is a phase 3, randomized trial of Pasireotide in 150 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.