This study will evaluate the safety and efficacy of two different doses of Pasireotide in patients with de novo or recurrent/persistent Cushing's Disease.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
162
Number of mUFC (Urinary Free Cortisol) Responders by Randomized Dose Group
A responder in the primary efficacy analysis was a patient with a mUFC≤ULN at Month 6 and whose dose was not increased prior to Month 6.
Time frame: 6 months
Change From Baseline in mUFC
Twenty four hour urine samples were collected to obtain mUFC measurements. A negative change from baseline indicates improvement.
Time frame: baseline, 3 months, 12 months
Time to First UFC Response
Time to first UFC response is defined as the number of months from baseline to first attainment of UFC response.
Time frame: 12 months
Percent Change From Baseline in Serum Cortisol
Blood samlpes were drawn to obtain serum cortisol levels. A negative change from baseline indicates improvement.
Time frame: baseline, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, 60, 63, 66, 69, 72, 75, 78 months
Percent Change From Baseline in Mean Adrenocorticotropic Hormone (ACTH)
Blood samples were drawn to obtain ACTH levels. A negative change from baseline indicates improvement.
Time frame: baseline, 0.5, 1, 1.5, 2, 2.5, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 15, 18, 21, 24, 27, 30, 33, 36, 39, 42, 45, 48, 51, 54, 57, 60, 63, 66, 69, 72, 75, 78 months
Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Sitting Sytolic Blood Pressure (SBP) and Sitting Diastolic Blood Pressure (DBP)
Sitting blood pressure assessments were performed at every study visit. A negative change from baseline indicates improvement.
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Stanford University Medical Center Stanford Cancer Center (3)
Stanford, California, United States
University Chicago Hospital Dept. of Univ of Chicago
Chicago, Illinois, United States
Dana Farber Cancer Institute The Melanoma Program
Boston, Massachusetts, United States
Columbia University Medical Center- New York Presbyterian Columbia University DeptofMed
New York, New York, United States
Cleveland Clinic Foundation Dept. of Cleveland Clinic (6)
Cleveland, Ohio, United States
Oregon Health & Sciences University Dept.ofOregonHealth&SciencesU.
Portland, Oregon, United States
University of Texas Southwestern Medical Center Clinical-TranslationalRes.Ctr.
Dallas, Texas, United States
University of Texas/MD Anderson Cancer Center Dept.ofMDAndersonCancerCtr(8)
Houston, Texas, United States
Baylor College of Medicine
Houston, Texas, United States
Swedish Medical Center Dept.ofSeattle Neuroscience(2)
Seattle, Washington, United States
...and 58 more locations
Time frame: baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60
Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Body Mass Index (BMI)
BMI was determined by using height and weight measurements. A negative change from baseline indicates improvement.
Time frame: baseline, month 3, month 6, month 12, month 24, month 36, month 48 and month 60
Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Waist Circumference
Waist circumference was measured with a measuring tape correctly positioned. A negative change from baseline indicates improvement.
Time frame: baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60
Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Total Cholesterol and Triglycerides
Blood samples were drawn to obtain total cholesterol and triglycerides' levels. A negative change from baseline indicates improvement.
Time frame: baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60
Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Beck Depression Inventory (BDI-II) Score
The BDI-II is a 21 item self-report rating inventory measuring characteristic attitudes and symptoms of depression. The BDI-II contains 21 questions, each answer being scored on a scale value of 0 to 3. Higher total scores indicate more severe depressive symptoms. The scores range as follows: 0-13: minimal depression; 14-19: mild depression; 20-28: moderate depression; and 29-63: severe depression. A negative change from baseline indicates imrpovement.
Time frame: baseline, month 3, month 6, month 12, month 18, month 24
Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Ferriman-Galway Hirsutism Score
The Ferriman Gallwey scoring system is used to score the degree of excess male pattern body hair. The scorecard of every body location under survey begins from 0 (no excessive terminal hair growth) to 4 (extensive terminal hair growth) and the numbers are added up to a maximum count of 36. A score \>= 6 indicates the hirsutism. A negative change from baseline indicates imrpovement.
Time frame: baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60
Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Bone Mineral Density (BMD)
BMD was measured using Lunar or Hologic dual-energy X-ray absorptiometry (DXA) Instruments. Measurements were done in the lumbar vertebrae (L1-L4), proximal femur (total hip) and proximal femur (femur neck). A negative change from baseline indicates imrpovement.
Time frame: baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60
Mean Change From Baseline in Clinical Signs and Symptoms of Cushing's Disease: Body Composition
Body composition as in percentage of body fat by region was assessed by total body scan. A negative change from baseline indicates improvement.
Time frame: baseline, month 3, month 6, month 12, month 24, month 36, month 48, month 60
Change From Baseline in Tumor Volume
Pituitary magnetic resonance imaging (MRI) was performed to determine tumor volume. A negative change from baseline indicates imrpovement.
Time frame: baseline, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60, 66, 72, 78 months
Percentage Change From Baseline in Health Related Quality of Life (HRQL) Score
A Cushing's syndrome health related quality of life (HRQL) questionnaire was completed. The Cushing's Syndrome HRQL questionnaire contains 12 sentences with 5 possible answers each. The answers are based on Likert scales, with 5 response categories: Always, Often, Sometimes, Rarely and Never; or Very much, Quite a bit, Somewhat, Very little, and Not at all. The answers to each of the items are rated on a scale of 1 to 5. "1" corresponds to the response category "Always" or "Very much" and "5" corresponds to the category "Never" or "Not at all". The score is the sum of all item responses and can range from 12 to 60 points. The lower the score, the greater the Cushing's Syndrome impacts on HRQoL. A positive change from baseline indicates improvement.
Time frame: baseline, 3 months, 6 months, 12 months