Efficacy and Safety Study of ACZ885 in Patients With Active Recurrent or Chronic TNF-receptor Associated Periodic Syndrome (TRAPS).

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01242813
First received: November 16, 2010
Last updated: January 5, 2016
Last verified: January 2016
Results First Received: November 2, 2015  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: TNF-receptor Associated Periodic Syndromes (TRAPS)
Intervention: Drug: ACZ885

  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
The study was conducted at 6 centers in 3 countries.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 29 participants were screened, out of which 20 were enrolled and exposed to study medication. Nine participants were considered as screening failures due to unacceptable laboratory value or test procedure results.

Reporting Groups
  Description
Canakinumab Participants received body-weight stratified dosage of canakinumab (2 milligram/ kilogram (mg/kg) for participants equal to or less than (≤) 40 kg or 150 mg for participants more than (>) 40 kg) through subcutaneous (s.c.) route as the starting dose at baseline and monthly for 4 months. The dose was escalated at Day 8 if dose of canakinumab was not sufficient to resolve the qualifying TNF-receptor associated periodic syndrome (TRAPS) flare.

Participant Flow:   Overall Study
    Canakinumab  
STARTED     20  
COMPLETED     18  
NOT COMPLETED     2  
Lost to Follow-up                 2  



  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.
The analysis was performed on Safety Set (SAF) defined as all participants who received at least one application of study treatment and had least one post-baseline safety assessment.

Reporting Groups
  Description
Canakinumab Participants received body-weight stratified dosage of canakinumab (2 mg/kg for participants ≤ 40 kg or 150 mg for participants > 40 kg) through s.c. route as the starting dose at baseline and monthly for 4 months. The dose was escalated at Day 8 if dose of canakinumab was not sufficient to resolve the qualifying TRAPS flare.

Baseline Measures
    Canakinumab  
Number of Participants  
[units: participants]
  20  
Age  
[units: Years]
Mean (Standard Deviation)
  34.62  (18.362)  
Gender  
[units: participants]
 
Female     7  
Male     13  
Region of Enrollment  
[units: participants]
 
Ireland     2  
Italy     10  
United Kingdom     8  



  Outcome Measures
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1.  Primary:   Percentage of Participants With Complete or Almost Complete Response at Day 15   [ Time Frame: Day 15 ]

2.  Secondary:   Percentage of Participants With Complete or Almost Complete Response at Day 8   [ Time Frame: Day 8 ]

3.  Secondary:   Percentage of Participants With Complete Clinical Remission at Day 8 and 15   [ Time Frame: Day 8 and Day 15 ]

4.  Secondary:   Percentage of Participant With Target Levels of C-reactive Protein (CRP) and Serum Amyloid A Protein (SAA) at Day 8 and 15   [ Time Frame: Day 8 and Day 15 ]

5.  Secondary:   Time to Physician’s Assessed Clinical Remission   [ Time Frame: Baseline up to Day 15 ]

6.  Secondary:   Percentage of Participants With Complete or Almost Complete Response at Day 15 After Receiving Additional Dose at Day 8   [ Time Frame: Day 15 ]

7.  Secondary:   Time to Participant's Assessed Clinical Remission   [ Time Frame: Baseline up to Day 15 ]

8.  Secondary:   Percentage Change From Baseline in C-reactive Protein (CRP) and Serum Amyloid A (SAA) Concentration to End of Study   [ Time Frame: Day 1 up to Day 953 (End of study) ]

9.  Secondary:   Percentage of Participants With Defined Grades for Skin Rash, Eye Manifestations, Extremity Pain and Abdominal Pain   [ Time Frame: Day 113 (end of treatment period) up to Day 925 (End of study) ]

10.  Secondary:   Percentage of Participants With Defined Grades in Physician’s Global Assessment Score   [ Time Frame: Day 1 up to Day 953 (End of study) ]

11.  Secondary:   Percentage of Participants With Defined Grades in Participant's Global Assessment Score   [ Time Frame: Day 1 up to Day 253 (End of follow-up period) ]

12.  Secondary:   Percentage of Relapsed Participants   [ Time Frame: Day 15, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449,477,505, 533, 561, 589, 617, 645, 673,701, 729,757, 785, 813, 841,869, 897, 925 and 953 ]

13.  Secondary:   Time to Relapse After Last Dose of Canakinumab   [ Time Frame: Day 85 to Day 253 (End of treatment period to Follow-up period) ]

14.  Secondary:   Percentage of Participants Who Relapsed and Received Rescue Medication   [ Time Frame: Day 85 to Day 953 (End of treatment period to End of study) ]

15.  Secondary:   Serum Concentration of Canakinumab   [ Time Frame: Day 3, 8, 15, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449, 533, 561, 589, 617, 645, 673, 729, 785, 841, 897, 925 and 953 ]

16.  Secondary:   Serum Concentration of Total Interleukin-1β Antibody (IL-1β)   [ Time Frame: Day 3, 8, 15, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449, 533, 561, 589, 617, 645, 673, 729, 785, 841, 897, 925 and 953 ]

17.  Secondary:   Number of Participants With Anti-canakinumab Antibodies at Any Visit   [ Time Frame: Day 1 up to Day 953 (End of study) ]


  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


No publications provided


Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01242813     History of Changes
Other Study ID Numbers: CACZ885D2203
2010-020061-24
Study First Received: November 16, 2010
Results First Received: November 2, 2015
Last Updated: January 5, 2016
Health Authority: United States: Food and Drug Administration
Ireland: The Food Safety Authority of Ireland
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Italy: AIFA-Italian Medicines Agency