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Safety and Efficacy of Different Dose Levels of Pasireotide in Patients With de Novo, Persistent or Recurrent Cushing's Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00434148
First received: February 9, 2007
Last updated: October 31, 2014
Last verified: October 2014
Results First Received: January 3, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Cushing's Disease
Intervention: Drug: Pasireotide

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
165 patients were randomized, however 1 patient from the 600 microgram group and 2 patients from the 900 microgram group were not treated.

Reporting Groups
  Description
600ug b.i.d All patients received a double blind dose of 600 µg subcutaneously ( s.c.) two times a day ( b.i.d) Pasireotide. Patients continued at this dose until Month 6 (double-blind treatment if their Month 3 mUFC was a ≤2 X ULN and b) below or equal to their baseline mUFC. Patients not meeting these mUFC criteria at Month 3 were unblinded and were required to increase their dose to 900 µg b.i.d, given on an open-label basis. These patients were classified as non-responders for the Month 6 Primary analysis. Patients who did not escalate to the 900 µg b.i.d dose were to be discontinued from the study.
900ug b.i.d All patients received a double blind dose of 900 µg subcutaneously ( s.c.) two times a day ( b.i.d) Pasireotide. Patients continued at this dose until Month 6 (double-blind treatment) if their Month 3 mUFC was a) ≤ 2 x ULN and b)below or equal to their baseline mUFC. Patients not meeting these mUFC criteria at Month 3 were unblended and were offered to increase their dose to 1200 µg b.i.d., given on an open-label basis. These patients were classified as non-responders for the Month 6 Primary analysis. Patients who did not escalate to the 1200 µg b.i.d dose were to be discontinued from the study. [China only: Patients in the 900 µg b.i.d dose group were not offered to have their dose increased to 1200 µg b.i.d but had to be discontinued.

Participant Flow:   Overall Study
    600ug b.i.d     900ug b.i.d  
STARTED     82 [1]   80  
Ongoing in Extension Phase     19     25  
COMPLETED     33     32  
NOT COMPLETED     49     48  
Adverse Event                 13                 15  
Lack of Efficacy                 19                 22  
Withdrawal by Subject                 13                 11  
Protocol Violation                 4                 0  
[1] "Started" indicates Full Analysis Set. Patients randomized and treated.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
600ug b.i.d No text entered.
900ug b.i.d No text entered.
Total Total of all reporting groups

Baseline Measures
    600ug b.i.d     900ug b.i.d     Total  
Number of Participants  
[units: participants]
  82     80     162  
Age  
[units: years]
Mean ± Standard Deviation
  40.5  ± 12.97     39.9  ± 10.77     40.2  ± 11.90  
Gender  
[units: participants]
     
Female     62     64     126  
Male     20     16     36  



  Outcome Measures

1.  Primary:   Proportion of mUFC (Urinary Free Cortisol) Responders by Randomized Dose Group   [ Time Frame: 6 months ]

2.  Secondary:   Reduction of UFC at Months 3 and 12   [ Time Frame: 3 and 12 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

3.  Secondary:   Time to First Response   [ Time Frame: Month 1, 2, 3, 4, 5, 6, 12 and every 3 months to study end. ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

4.  Secondary:   Assessment of Plasma ACTH and Serum Cortisol Level   [ Time Frame: Month 1, 2, 3, 4, 5, 6, 12 and every 3 months to study end. ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No

5.  Secondary:   Assessment of Clinical Signs and Symptoms   [ Time Frame: Month 1, 2, 3, 4, 5, 6, 12 and every 3 months to study end. ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862-778-8300


No publications provided by Novartis

Publications automatically indexed to this study:

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00434148     History of Changes
Other Study ID Numbers: CSOM230B2305, 2006-004111-22
Study First Received: February 9, 2007
Results First Received: January 3, 2013
Last Updated: October 31, 2014
Health Authority: United States: Food and Drug Administration
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Belgium: The Federal Public Service (FPS) Health, Food Chain Safety and Environment
Brazil: National Health Surveillance Agency
Canada: Health Canada
China: Ministry of Health
Denmark: Danish Medicines Agency
Finland: Finnish Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: German Institute of Medical Documentation and Information
Greece: National Organization of Medicines
Italy: The Italian Medicines Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Portugal: National Pharmacy and Medicines Institute
Spain: Ministry of Health and Consumption
Turkey: Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency