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Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis
ClinicalTrials.gov Identifier:
NCT00685373
First received: May 27, 2008
Last updated: July 24, 2012
Last verified: July 2012
History of Changes
Results First Received: April 25, 2011  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Cryopyrin-associated Periodic Syndromes:
Familial Cold Autoinflammatory Syndrome
Muckle Wells Syndrome
Neonatal Onset Multisystem Inflammatory Disease
Intervention: Drug: Canakinumab (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
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
This study included participants previously enrolled in CACZ885A2102 (NCT00487708), CACZ885D2304 (NCT00465985) and ACZ885 naive patients.

Reporting Groups
  Description
Canakinumab (ACZ885) Subcutaneous injection every 8 weeks based on participant's body weight. Body weight >40 kilogram (kg): 150 milligrams (mg) per injection and body weight <= 40 kg: 2 mg/kg per injection. For participants who did not experience sufficient symptomatic relief, an up-titration to the dose and/or more frequent doses were permitted as per protocol.

Participant Flow:   Overall Study
    Canakinumab (ACZ885)  
STARTED     166  
COMPLETED     151  
NOT COMPLETED     15  
Adverse Event                 3  
Withdrawal by Subject                 5  
Lack of Efficacy                 3  
Condition no longer requires study drug                 1  
Lost to Follow-up                 2  
Protocol Violation                 1  



  Baseline Characteristics
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Reporting Groups
  Description
Canakinumab (ACZ885) Subcutaneous injection every 8 weeks based on participant's body weight. Body weight >40 kilogram (kg): 150 milligrams (mg) per injection and body weight <= 40 kg: 2 mg/kg per injection. For participants who did not experience sufficient symptomatic relief, an up-titration to the dose and/or more frequent doses were permitted as per protocol.

Baseline Measures
    Canakinumab (ACZ885)  
Number of Participants  
[units: participants]
 		  166  
Age, Customized  
[units: participants]
 		 
>= 3 to < 18 years     47  
>= 18 to < 41 years     65  
>= 41 to < 75 years     52  
>= 75 years     2  
Gender  
[units: participants]
 		 
Female     97  
Male     69  
Disease Characteristics  
[units: participants]
 		 
Muckle-Wells Syndrome     103  
Familial Cold Autoinflammatory Syndrome     30  
Neonatal Onset Multi-System Inflammatory Syndrome     32  
Information Missing     1  



  Outcome Measures
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1.  Primary:   The Number of Participants With Adverse Events (AEs), Death, Serious Adverse Events (SAEs), Discontinuation of Study Drug Due to an AE, Infections and Infestations and Injection Site Reactions   [ Time Frame: 2 years depending on when the participant enters the study ]
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Measure Type Primary
Measure Title The Number of Participants With Adverse Events (AEs), Death, Serious Adverse Events (SAEs), Discontinuation of Study Drug Due to an AE, Infections and Infestations and Injection Site Reactions
Measure Description The number of participants with Adverse Events and Infections & Infestations are regardless of study drug relationship by primary system organ class preferred term equal and/or greater than 2% in any group. The number of participants with mild injection site reactions= mild reactions observed on at least one occasion but no moderate or severe reactions. The number of participants with moderate injection site reactions= moderate reactions observed on at least one occasion but no severe reactions.
Time Frame 2 years depending on when the participant enters the study  
Safety Issue Yes  

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.
Safety Population defined as all participants who received at least one dose of study drug.

Reporting Groups
  Description
Canakinumab (ACZ885) Subcutaneous injection every 8 weeks based on participant's body weight. Body weight >40 kilogram (kg): 150 milligrams (mg) per injection and body weight <= 40 kg: 2 mg/kg per injection. For participants who did not experience sufficient symptomatic relief, an up-titration to the dose and/or more frequent doses were permitted as per protocol.

Measured Values
    Canakinumab (ACZ885)  
Number of Participants Analyzed  
[units: participants]
 		  166  
The Number of Participants With Adverse Events (AEs), Death, Serious Adverse Events (SAEs), Discontinuation of Study Drug Due to an AE, Infections and Infestations and Injection Site Reactions  
[units: participants]
 		 
Adverse Event     150  
Death     0  
Serious Adverse Event     18  
Discontinuation of study drug due to an AE     4  
Infections and Infestations     109  
Any Injection Site Reactions     13  
Mild Injection Site Reactions     11  
Moderate Injection Site Reactions     2  

No statistical analysis provided for The Number of Participants With Adverse Events (AEs), Death, Serious Adverse Events (SAEs), Discontinuation of Study Drug Due to an AE, Infections and Infestations and Injection Site Reactions



2.  Secondary:   The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Inflammation Markers.   [ Time Frame: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years ]
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3.  Secondary:   Immunogenicity of Canakinumab (ACZ885)   [ Time Frame: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years ]
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4.  Secondary:   Pharmacokinetics   [ Time Frame: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years ]
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  Serious Adverse Events
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  Other Adverse Events
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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.


Limitations and Caveats
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.  


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
ClinicalTrials.gov Identifier: NCT00685373     History of Changes
Other Study ID Numbers: CACZ885D2306
Study First Received: May 27, 2008
Results First Received: April 25, 2011
Last Updated: July 24, 2012
Health Authority: United States: Food and Drug Administration
Germany: Paul-Ehrlich-Institut
Spain: Spanish Agency of Medicines
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Italy: The Italian Medicines Agency
Turkey: Ministry of Health
India: Drugs Controller General of India