Extension Study to Assess the Safety and Efficacy of Pasireotide in Participants With Cushing's Disease

What Was Tested

The trial (phase 2) is built around Pasireotide.

Cushing's disease is a rare serious condition that is caused by an adrenocorticotropic hormone (ACTH) secreting pituitary adenoma.

It had 1 arm (groups): one (experimental).

The registry classes the interventions as drug.

Who Took Part

19 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: The participant did not experience any unacceptable adverse events of tolerability issues during the original 15 day treatment.

The full record lists 3 inclusion and 3 exclusion criteria; among the exclusions: participant who have developed poorly controlled diabetes mellitus as indicated by ketoacidosis or hemoglobin (hgb) a1c (hgba1c) \> 10 since starting \[study csom230b2208\].

How the Study Was Run

The set-up was single-group and open-label.

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

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

It started in 2004-08-13 with completion listed for 2013-07-08.

It ran at 8 sites in United States (3), Germany (2), France (1), Italy (1) and United Kingdom (1).

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits (time frame: Month 6). Change From Baseline in Mean Urinary Free Cortisol (UFC) (time frame: Core Baseline, Days 14/15 (Core study), Months 6, 12, 24 and 102).

It also tracked 5 secondary outcomes, for example Number of Participants Who Had At Least One Adverse Event (AE), Change From Baseline in Serum Cortisol Levels, Plasma Trough Concentrations (Ctrough) of Pasireotide in UFC Responders, Change From Baseline in Plasma Adrenocorticotropic Hormone (ACTH) Levels and Change From Baseline in Gene-expression and Protein in Blood and Urine for Biomarker Development.

What This Means in Plain Language

Put simply, this is a phase 2 trial of Pasireotide in 19 people.

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

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.