Long Term Safety and Efficacy of Pasireotide s.c. in Patients With Cushing's Disease

Sponsor

RECORDATI GROUP (Industry)

Overall Status

Recruiting

CT.gov ID

NCT02310269

Collaborator

(none)

200

Enrollment

Locations

133.1

Anticipated Duration (Months)

2.8

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a non-interventional, multinational, multi-center post-marketing study, to further document the safety and efficacy of pasireotide s.c. administered in routine clinical practice in patients with Cushing's disease. Patients with Cushing's disease and treated with pasireotide s.c. alone and in combination with other therapies will be monitored. For this study, each enrolled patient will be followed up for 3 years after enrollment. Patients who permanently discontinue pasireotide s.c. prior to completing the 3-year observation period will be followed up for 3 months after the last dose of pasireotide s.c.

Condition or Disease	Intervention/Treatment	Phase
Cushings Disease	Drug: SOM230

Study Design

Study Type:

Observational

Anticipated Enrollment :

200 participants

Observational Model:

Other

Time Perspective:

Prospective

Official Title:

Non-interventional Study for the Generation of Long Term Safety and Efficacy Data of Pasireotide s.c. in Patients With Cushing's Disease (Post-Authorization Safety Study)

Actual Study Start Date :

Mar 28, 2013

Anticipated Primary Completion Date :

Sep 27, 2023

Anticipated Study Completion Date :

May 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Pasireotide	Drug: SOM230

Arm

Intervention/Treatment

Pasireotide

Drug: SOM230

Outcome Measures

Primary Outcome Measures

Number of Participants with Adverse Events as a Measure of Safety and Tolerability profile of pasireotide s.c. [3-year follow-up]
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

Secondary Outcome Measures

The proportion of patients that reach mean UFC ≤ ULN as short and long-term efficacy measure [At 1, 3, 6, 12, 24 and 36 months after enrolling into the study]
The proportion of patients with a mean UFC ≤ ULN at 1, 3, 6, 12, 24 and 36 months after enrolling into the study
Changes of mean UFC measures of disease activity [3-year follow-up]
The absolute and percentage change from baseline of mean UFC measures of disease activity after enrolling into the study.
Proportion of patients achieving serum cortisol Normalization of disease activity [3-year follow-up]
Proportion of patients achieving serum cortisol Normalization of disease activity where normalization refers to being within the upper and lower limit of normal ranges
The absolute and percentage change of blood pressure from baseline [3-year follow-up]
The absolute and percentage change of blood pressure from baseline after enrolling into the study
The proportion of patients with favorable shift of blood pressure from baseline [3-year follow-up]
The proportion of patients with favorable shift of blood pressure from baseline after enrolling into the study
Changes in safety and efficacy parameters over a period of 3 months [3 months after patient's discontinuation]
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, γGT, 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.
The absolute and percentage change in tumor size [3-year follow-up]
The absolute and percentage change in tumor size after enrolling into the study
The absolute and percentage change from baseline in patient-reported outcome questionnaires [3-year follow-up]
The absolute and percentage change from baseline in patient -reported outcome questionnaires (Cushing QoL and EURO QoL) after enrolling into the study
Change of serum cortisol measures of disease activity [3-year follow-up]
The absolute and percentage change from baseline of serum cortisol measures of disease activity after enrolling into the study
Change of serum cortisol after dexamathasone testing measures of disease activity [3-year followup]
The absolute and percentage change from baseline of serum cortisol after dexamathasone testing measures of disease activity after enrolling into the study
Changes of salivary cortisol measures of disease activity [3-year follow-up]
The absolute and percentage change from baseline of salivary cortisol measures of disease activity after enrolling into the study
Changes of ACTH measures of disease activtity [3-year follow-up]
The absolute and percentage change from baseline of ACTH measures of disease activity after enrolling into the study
Proportion of patients achieving serum cortisol after dexamethasone testing Normalization of disease activity [3-year follow-up]
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
Proportion of patients achieving salivary cortisol Normalization of disease activity [3-year follow-up]
Proportion of patients achieving salivary cortisol of disease activity where normalization refers to being within the upper and lower limit of normal ranges
Proportion of patients achieving ACTH Normalization of disease activity [3-year follow-up]
Proportion of patients achieving ACTH of disease activity where normalization refers to being within the upper and lower limit of normal ranges
Changes of fasting serum lipid profile measures of disease activtity [3-year follow-up]
The absolute and percentage change from baseline of fasting serum lipid profile measures of disease activity after enrolling into the study
The absolute and percentage change of body weight from baseline [3-year follow-up]
The absolute and percentage change of body weight from baseline after enrolling into the study
The absolute and percentage change of body mass index from baseline [3-year follow-up]
The absolute and percentage change of body mass index from baseline after enrolling into the study
The absolute and percentage change of waist circumference from baseline [3-year follow-up]
The absolute and percentage change of waist circumference from baseline after enrolling into the study
The proportion of patients with favorable shift of body weight from baseline [3-year follow-up]
The proportion of patients with favorable shift of body weight from baseline after enrolling into the study
The proportion of patients with favorable shift of body mass index from baseline [3-year follow-up]
The proportion of patients with favorable shift of body mass index from baseline after enrolling into the study
The proportion of patients with favorable shift of waist circumference from baseline [3-year follow-up]
The proportion of patients with favorable shift of waist circumference from baseline after enrolling into the study

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male or female patients aged 18 years or older with a diagnosis of Cushing's disease for whom surgery has failed or for whom surgery is not an option
Patients must be treated with pasireotide s.c. started either at the first visit for this study or prior to study entry

Exclusion Criteria:

Patients with ectopic ACTH-dependent Cushing's syndrome
Patients with adrenal Cushing's syndrome
Patients with Pseudo Cushing's syndrome

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	St Josephs Hospital and Medical Center	Phoenix	Arizona	United States	85013
2	University of Southern California CSOM230B2410 - SC	Los Angeles	California	United States	90033
3	Diabetes Research Institute SC	Miami	Florida	United States	22136
4	LifeBridge Heath Cardiovascular Institute, Sinai Hospital SC - SOM230B2410	Baltimore	Maryland	United States	21215
5	Massachusetts General Hospital SC - SOM230B2410	Boston	Massachusetts	United States	02114
6	Ohio State University SC - SOM230B2410	Columbus	Ohio	United States	43210
7	Endocrinology Services	Bend	Oregon	United States	97702
8	Allegheny Endocrinology Associates	Pittsburgh	Pennsylvania	United States	15212
9	R. I. Hospital Medical Oncology Clinical Research SC	Providence	Rhode Island	United States	02903
10	Swedish Medical Center Dept.ofSeattle Neuroscience(2)	Seattle	Washington	United States	98122-4379
11	Recordati Investigative Site	Montreal	Quebec	Canada	H2W 1T8
12	Recordati Investigative Site	Sherbrooke	Quebec	Canada	J1H 5N4
13	Recordati Investigative Site	Quebec		Canada	G1V 4G2
14	Recordati Investigative Site	Cali		Colombia
15	Recordati Investigative Site	Amiens Cedex 1		France	80054
16	Recordati Investigative Site	Bron Cedex		France	69677
17	Recordati Investigative Site	Dijon		France	21000
18	Recordati Investigative Site	Lille Cedex		France	59037
19	Recordati Investigative Site	Marseille		France	13385
20	Recordati Investigative Site	Montpellier Cedex 5		France	34295
21	Recordati Investigative Site	Paris		France	75014
22	Recordati Investigative Site	Reims		France	51092
23	Recordati Investigative Site	Saint Mande		France	94160
24	Recordati Investigative Site	St Priest en Jarez Cedex		France	42277
25	Recordati Investigative Site	Strasbourg		France	67098
26	Recordati Investigative Site	Mannheim	Baden-Wuerttemberg	Germany	68305
27	Recordati Investigative Site	Aachen		Germany	52074
28	Recordati Investigative Site	Berlin		Germany	10117
29	Recordati Investigative Site	Berlin		Germany	13353
30	Recordati Investigative Site	Duesseldorf		Germany	40225
31	Recordati Investigative Site	Erlangen		Germany	91054
32	Recordati Investigative Site	Essen		Germany	45147
33	Recordati Investigative Site	Frankfurt		Germany	60590
34	Recordati Investigative Site	Frankfurt		Germany	60596
35	Recordati Investigative Site	Freiburg		Germany	79106
36	Recordati Investigative Site	Halle		Germany	06108
37	Recordati Investigative Site	Hamburg		Germany	20246
38	Recordati Investigative Site	Hamburg		Germany	22587
39	Recordati Investigative Site	Hannover		Germany	30159
40	Recordati Investigative Site	Hannover		Germany	30625
41	Recordati Investigative Site	Leipzig		Germany	04103
42	Recordati Investigative Site	Magdeburg		Germany	39120
43	Recordati Investigative Site	Marburg		Germany	35039
44	Recordati Investigative Site	Muenchen		Germany	81377
45	Recordati Investigative Site	Muenchen		Germany	81667
46	Recordati Investigative Site	Muenster		Germany	48149
47	Recordati Investigative Site	Oldenburg		Germany	26122
48	Recordati Investigative Site	Wuerzburg		Germany	97080
49	Recordati Investigative Site	Petach Tikva		Israel	49100
50	Recordati Investigative Site	Tel Aviv		Israel	6423906
51	Recordati Investigative Site	Ancona	AN	Italy	60126
52	Recordati Investigative Site	Messina	ME	Italy	98125
53	Recordati Investigative Site	Milano	MI	Italy	20122
54	Recordati Investigative Site	Milano	MI	Italy	20149
55	Recordati Investigative Site	Palermo	PA	Italy	90127
56	Recordati Investigative Site	Padova	PD	Italy	35128
57	Recordati Investigative Site	Pisa	PI	Italy	56124
58	Recordati Investigative Site	Torino	TO	Italy	10126
59	Recordati Investigative Site	Napoli		Italy	80131
60	Recordati Investigative Site	Padova		Italy	35128
61	Recordati Investigative Site	El Achrafiyé		Lebanon	166830
62	Recordati Investigative Site	Maastricht	AZ	Netherlands	5800
63	Recordati Investigative Site	Nijmegen		Netherlands	6500 HB
64	Recordati Investigative Site	Brasov		Romania
65	Recordati Investigative Site	Bucuresti		Romania	011863
66	Recordati Investigative Site	Cluj		Romania	400006
67	Recordati Investigative Site	Târgu-Mureş		Romania	540061
68	Recordati Investigative Site	Gothenburg		Sweden	SE-413 45
69	Recordati Investigative Site	Uppsala		Sweden	SE-751 85
70	Recordati Investigative Site	Plymouth		United Kingdom	PL6 8DH
71	Recordati Investigative Site	Southampton		United Kingdom	SO16 6YD