An Open-label, Multi-center, Expanded Access Study of Pasireotide s.c. in Patients With Cushing's Disease.

What Was Tested

This registered phase 3 trial tested Pasireotide.

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

The registry classes the interventions as drug.

More on the Plan

Purpose of this study was to give access to pasireotide sc for patients with Cushing's disease as no medical treatment for Cushing's disease was approved at the time of the study initiation.

A confirmed Cushing's disease diagnosis was required.

Who Took Part

Enrollment: 104 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 any screening procedures. Male or female patients aged 18 years or greater. Karnofsky performance status \>60 (i.e. requires occasional assistance, but is able to care for most of his personal needs). For patients on previous medical treatment for Cushing's disease the following washout periods must be completed before screening assessments are performed. Inhibitors of steroidogenesis (e.g. ketoconazole, metyrapone, rosiglitazone): 1 week.

The full record lists 13 inclusion and 25 exclusion criteria; among the exclusions: radiotherapy of the pituitary \<4 weeks before screening or patient who has not recovered from side effects; 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

It was run as a non-randomized, single-group and open-label study.

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 2011-08-16 with completion listed for 2017-01-26.

It ran at 65 sites in United States (17), Germany (16), Spain (8), Brazil (7), Greece (4) and Romania (4).

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Percentage of Patients With a Drug-related Adverse Event That is Recorded as Grade 3 or 4 or as a Serious Adverse Event (SAE) (time frame: Baseline up to approximately 256 weeks).

It also tracked 13 secondary outcomes, for example Percentage of Patients With Mean Urinary Free Cortisol (UFC) ≤ Upper Limit of Normal (ULN), Percent Change in Cushing Quality of Life and Work Productivity and Activity Impairment-General Health (WPAI-GH) Scores, Percent Change in Cushing's Disease Clinical Signs and Symptoms - Blood Pressure (BP), Percent Change in Cushing's Disease Clinical Signs and Symptoms - Pulse, Percent Change in Cushing's Disease Clinical Signs and Symptoms - Temperature and Percent Change in Cushing's Disease Clinical Signs and Symptoms - Body Mass Index (BMI).

What This Means in Plain Language

Put simply, this is a phase 3, non-randomized trial of Pasireotide in 104 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.