Cushing's disease is a rare serious condition that is caused by an adrenocorticotropic hormone (ACTH) secreting pituitary adenoma. This study assessed the long-term safety and efficacy of pasireotide in participants with Cushing's disease.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
19
Pasireotide 600 μg or 900 μg was administered as an SC injection.
Massachusetts General Hospital
Boston, Massachusetts, United States
Oregon Health & Sciences University Dept.ofOregonHealth&SciencesU.
Portland, Oregon, United States
University of Pennsylvania Medical Center
Philadelphia, Pennsylvania, United States
Novartis Investigative Site
Paris, France
Percentage of Responders With Mean Urinary Free Cortisol (UFC) Within Normal Limits
A participant was considered a responder if the mean UFC from the two 24-hour urine samples collected at Month 6 was within normal limits. The normal range for UFC is 55 to 276 nmol/day.
Time frame: Month 6
Change From Baseline in Mean Urinary Free Cortisol (UFC)
24-hour urine samples were collected to obtain mean UFC measurements. A negative mean change from baseline indicates improvement.
Time frame: Core Baseline, Days 14/15 (Core study), Months 6, 12, 24 and 102
Number of Participants Who Had At Least One Adverse Event (AE)
An AE was any undesirable sign, symptom or medical condition occurring after starting study drug even if the event is not considered to be related to study drug. AEs were assessed according to incident dose group: Pasireotide 1200 μg sc total daily dose (TDD), Pasireotide 1800 μg SC TDD and Pasireotide SC Any Dose. The incident dose for an AE was the last total daily dose administered on or prior to the AE onset date.
Time frame: Up to approximately 106 months
Change From Baseline in Serum Cortisol Levels
Blood samples were withdrawn to obtain the serum cortisol levels. A negative change from baseline indicates improvement.
Time frame: Core Baseline, Day 15 (Core study), Months 6, 12, 24, and Month 105 (end of the study)
Plasma Trough Concentrations (Ctrough) of Pasireotide in UFC Responders
Participants with Cushing's disease were considered responders if mean UFC levels from the 24-hour urine collections at Day 15 (Core study) and at extension Month 6, were within normal limits. Ctrough levels of pasireotide were measured at Day 15 of Core study and Month 6.
Time frame: Day 15 (Core study) and Month 6
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Novartis Investigative Site
Essen, Germany
Novartis Investigative Site
München, Germany
Novartis Investigative Site
Ancona, AN, Italy
Novartis Investigative Site
Belfast, United Kingdom
Change From Baseline in Plasma Adrenocorticotropic Hormone (ACTH) Levels
Blood samples were withdrawn to obtain the ACTH levels. A negative change from baseline indicates improvement.
Time frame: Core Baseline, Day 15 (Core study), Months 6, 12, 24 and Month 105 (end of the study)
Change From Baseline in Gene-expression and Protein in Blood and Urine for Biomarker Development
Time frame: Baseline to end of the study