Study of the Efficacy and Safety of Pasireotide s.c. +/- Cabergoline in Patients With Cushing's Disease

What Was Tested

This registered phase 2 trial tested Pasireotide.

It had 1 arm (groups): pasireotide +/- cabergoline (experimental).

Pasireotide alone or with cabergoline.

The registry classes the interventions as drug.

More on the Plan

This was an open-label, multi-center, international, non-comparative study with adult patients with confirmed diagnosis of Cushing's disease.

Given the fact that CD patients may need a multimodality treatment approach, the trial design aimed to mimic CD treatment by using a medical stepwise approach.

Therefore, the whole patient population started treatment with Pasireotide and only in patients within this population who did not achieve biochemical control, cabergoline was added.

The whole patient population had never received pasireotide or had received it in the past (reasons of discontinuation not related to safety).

Who Took Part

The registry lists 68 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: Written informed consent obtained prior to screening procedures. Adult patients with confirmed diagnosis of ACTH-dependent Cushing's disease as evidenced by all of the following:. The mean of three 24-hour urine samples collected within 2 weeks \> 1xULN with 2 out of 3 samples \>ULN. Morning plasma ACTH within the normal or above normal range. Male or female patients aged 18 years or greater.

The full record lists 17 inclusion and 24 exclusion criteria; among the exclusions: patients with compression of the optic chiasm that caused any visual field defect that required surgical intervention; patients with cushing's syndrome due to ectopic acth secretion; patients with hypercortisolism secondary to adrenal tumors or nodular (primary) bilateral adrenal hyperplasia.

How the Study Was Run

Design: single-group and open-label.

Being open-label, the Pasireotide study did not mask who received what.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2014-03-06 with completion listed for 2016-09-05.

It ran at 29 sites in Brazil (4), India (3), Turkey (Türkiye) (3), United States (2), Belgium (2) and Greece (2).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Percentage of Responders With Mean Urinary Free Cortisol (mUFC) ≤ 1.0xULN Collected or Imputed at Week 35 (time frame: Baseline up to week 35).

It also tracked 21 secondary outcomes, for example Mean Urinary Free Cortisol (mUFC) at Scheduled Visits, Percentage of Responders With Mean Urinary Free Cortisol (mUFC) ≤ 1.0xULN, Duration (Weeks) of Controlled or Partially Controlled Response, Plasma Adrenocorticotropic Hormone (ACTH), Serum Cortisol Levels and Sitting Systolic Blood Pressure at Week 35.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Pasireotide in 68 people.

It ended early (terminated); results are posted on the registry, and this page does not restate them.

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