Outcome
| Type |
Measure |
Description |
Time frame |
Safety issue |
| Primary |
Number of Participants with Adverse Events as a Measure of Safety and Tolerability profile of pasireotide s.c. |
Number of pasireotide s.c related adverse events and serious adverse events, when administered as monotherapy or in combination with other therapies in patients with Cushing's disease |
3-year follow-up |
|
| Secondary |
The proportion of patients that reach mean UFC = ULN as short and long-term efficacy measure |
The proportion of patients with a mean UFC = ULN at 1, 3, 6, 12, 24 and 36 months after enrolling into the study |
At 1, 3, 6, 12, 24 and 36 months after enrolling into the study |
|
| Secondary |
Changes of mean UFC measures of disease activity |
The absolute and percentage change from baseline of mean UFC measures of disease activity after enrolling into the study. |
3-year follow-up |
|
| Secondary |
Proportion of patients achieving serum cortisol Normalization of disease activity |
Proportion of patients achieving serum cortisol Normalization of disease activity where normalization refers to being within the upper and lower limit of normal ranges |
3-year follow-up |
|
| Secondary |
The absolute and percentage change of blood pressure from baseline |
The absolute and percentage change of blood pressure from baseline after enrolling into the study |
3-year follow-up |
|
| Secondary |
The proportion of patients with favorable shift of blood pressure from baseline |
The proportion of patients with favorable shift of blood pressure from baseline after enrolling into the study |
3-year follow-up |
|
| Secondary |
Changes in safety and efficacy parameters over a period of 3 months |
Safety parameters include: Adverse events , Serious Adverse Events, vital signs, blood pressure, heart rate, body temperature, blood glucose (fasting plasma glucose, HemoglobinA1c), hormones (IGF-1, GH, TSH/free T4), liver enzymes (AST, ALT, alkaline phosphatase, ?GT, total bilirubin), hematology, electrolytes, immunological events (e.g., allergic reactions: rash, pruritus, injection site reactions), gallbladder ultrasound and ECGs. Efficacy parameters include: Urinary Free Cortisol, serum cortisol, serum cortisol after dexamethasone testing, salivary cortisol, plasma ACTH, fasting serum lipid profile, Blood pressure, body weight, body mass index, waist circumference, tumor size and quality of life. |
3 months after patient's discontinuation |
|
| Secondary |
The absolute and percentage change in tumor size |
The absolute and percentage change in tumor size after enrolling into the study |
3-year follow-up |
|
| Secondary |
The absolute and percentage change from baseline in patient-reported outcome questionnaires |
The absolute and percentage change from baseline in patient -reported outcome questionnaires (Cushing QoL and EURO QoL) after enrolling into the study |
3-year follow-up |
|
| Secondary |
Change of serum cortisol measures of disease activity |
The absolute and percentage change from baseline of serum cortisol measures of disease activity after enrolling into the study |
3-year follow-up |
|
| Secondary |
Change of serum cortisol after dexamathasone testing measures of disease activity |
The absolute and percentage change from baseline of serum cortisol after dexamathasone testing measures of disease activity after enrolling into the study |
3-year followup |
|
| Secondary |
Changes of salivary cortisol measures of disease activity |
The absolute and percentage change from baseline of salivary cortisol measures of disease activity after enrolling into the study |
3-year follow-up |
|
| Secondary |
Changes of ACTH measures of disease activtity |
The absolute and percentage change from baseline of ACTH measures of disease activity after enrolling into the study |
3-year follow-up |
|
| Secondary |
Proportion of patients achieving serum cortisol after dexamethasone testing Normalization of disease activity |
Proportion of patients achieving serum cortisol after dexamethasone testing Normalization of disease activity where normalization refers to being within the upper and lower limit of normal ranges |
3-year follow-up |
|
| Secondary |
Proportion of patients achieving salivary cortisol Normalization of disease activity |
Proportion of patients achieving salivary cortisol of disease activity where normalization refers to being within the upper and lower limit of normal ranges |
3-year follow-up |
|
| Secondary |
Proportion of patients achieving ACTH Normalization of disease activity |
Proportion of patients achieving ACTH of disease activity where normalization refers to being within the upper and lower limit of normal ranges |
3-year follow-up |
|
| Secondary |
Changes of fasting serum lipid profile measures of disease activtity |
The absolute and percentage change from baseline of fasting serum lipid profile measures of disease activity after enrolling into the study |
3-year follow-up |
|
| Secondary |
The absolute and percentage change of body weight from baseline |
The absolute and percentage change of body weight from baseline after enrolling into the study |
3-year follow-up |
|
| Secondary |
The absolute and percentage change of body mass index from baseline |
The absolute and percentage change of body mass index from baseline after enrolling into the study |
3-year follow-up |
|
| Secondary |
The absolute and percentage change of waist circumference from baseline |
The absolute and percentage change of waist circumference from baseline after enrolling into the study |
3-year follow-up |
|
| Secondary |
The proportion of patients with favorable shift of body weight from baseline |
The proportion of patients with favorable shift of body weight from baseline after enrolling into the study |
3-year follow-up |
|
| Secondary |
The proportion of patients with favorable shift of body mass index from baseline |
The proportion of patients with favorable shift of body mass index from baseline after enrolling into the study |
3-year follow-up |
|
| Secondary |
The proportion of patients with favorable shift of waist circumference from baseline |
The proportion of patients with favorable shift of waist circumference from baseline after enrolling into the study |
3-year follow-up |
|
