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Safety, Efficacy and PK/PD of QGE031 vs. Placebo in Patients With Active Bullous Pemphigoid Despite Oral Steroid Treatment

This study has been terminated.
(This study was stopped after Part 1 completed and was terminated because the predefined criteria of efficacy was not reached ( >50% better then placebo))
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01688882
First received: September 17, 2012
Last updated: March 28, 2016
Last verified: March 2016
Results First Received: March 28, 2016  
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: Bullous Pemphigoid
Interventions: Drug: QGE031
Drug: Placebo

  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
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Reporting Groups
  Description
QGE031 QGE031 240 mg Q2W s.c.
Placebo Placebo to Match Q2W s.c.

Participant Flow:   Overall Study
    QGE031     Placebo  
STARTED     13     7 [1]
Safety Follow-up     11     5  
COMPLETED     10     6 [2]
NOT COMPLETED     3     1  
Adverse Event                 1                 0  
Unsatisfactory therapeutic effect                 1                 1  
Administrative problems                 1                 0  
[1] 4 patients who completed 12 weeks of placebo changed to open-label QGE031 treatment.
[2] The one patient that discontinued was already switched to open-label QGE031 treatment.



  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
QGE031 QGE031 240 mg Q2W s.c.
Placebo Placebo to Match Q2W s.c.
Total Total of all reporting groups

Baseline Measures
    QGE031     Placebo     Total  
Number of Participants  
[units: participants]
  13     7     20  
Age  
[units: years]
Mean (Standard Deviation)
  63.7  (14.85)     67.9  (14.60)     65.2  (14.52)  
Gender  
[units: participants]
     
Female     3     4     7  
Male     10     3     13  



  Outcome Measures
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1.  Primary:   Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12   [ Time Frame: 12 weeks ]

2.  Secondary:   Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks   [ Time Frame: 6 weeks ]

3.  Secondary:   Number of Patients Investigator Global Assessment Score Over 12 Weeks   [ Time Frame: Baseline (week 0), week 6 and week 12 ]


  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
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  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



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01688882     History of Changes
Other Study ID Numbers: CQGE031X2202
Study First Received: September 17, 2012
Results First Received: March 28, 2016
Last Updated: March 28, 2016
Health Authority: United States: Food and Drug Administration
Austria: Agency for Health and Food Safety
Germany: Paul-Ehrlich-Institut
Taiwan: Center for Drug Evaluation
France: Ministry of Health