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Trial record 4 of 16 for:    "Muckle-Wells syndrome"

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

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ClinicalTrials.gov Identifier: NCT00685373
Recruitment Status : Completed
First Posted : May 28, 2008
Results First Posted : May 27, 2011
Last Update Posted : November 4, 2016
Sponsor:
Information provided by (Responsible Party):
Novartis

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (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)
Enrollment 166
Recruitment Details  
Pre-assignment Details This study included participants previously enrolled in CACZ885A2102 (NCT00487708), CACZ885D2304 (NCT00465985) and ACZ885 naive patients.
Arm/Group Title Canakinumab (ACZ885)
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 166
Completed 151
Not Completed 15
Reason Not Completed
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
Arm/Group Title Canakinumab (ACZ885)
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 166
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 166 participants
>= 3 to < 18 years 47
>= 18 to < 41 years 65
>= 41 to < 75 years 52
>= 75 years 2
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 166 participants
Female
97
  58.4%
Male
69
  41.6%
Disease Characteristics  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 166 participants
Muckle-Wells Syndrome 103
Familial Cold Autoinflammatory Syndrome 30
Neonatal Onset Multi-System Inflammatory Syndrome 32
Information Missing 1
1.Primary Outcome
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
Hide 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
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety Population defined as all participants who received at least one dose of study drug.
Arm/Group Title Canakinumab (ACZ885)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 166
Measure Type: Number
Unit of Measure: 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
2.Secondary Outcome
Title 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.
Hide Description

Disease relapse following complete response is defined as inflammation markers: C-Reactive Protein (CRP) and/or Serum Amyloid A (SAA) result > 30 mg/L AND Physician's Global Assessment of Autoinflammatory Disease Activity > minimal or Physician's Global Assessment >= minimal AND Skin Disease Assessment > minimal.

Physician's Global Assessment of Autoinflammatory Disease Activity and Skin Disease Assessment (urticarial skin rash) are completed by the investigator using a 5 point rating scale: absent, minimal, mild, moderate and severe.

Time Frame Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety Population defined as all participants who had at least one dose of study drug and were included in the Relapse Assessment.
Arm/Group Title Canakinumab (ACZ885)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 141
Measure Type: Number
Unit of Measure: Percentage of participants
90.1
3.Secondary Outcome
Title Immunogenicity of Canakinumab (ACZ885)
Hide Description The number of participants who tested positive for anti-ACZ885 antibodies using the Biacore Assay at the end of the study.
Time Frame Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety Population defined as all participants who received at least one dose of study drug and were tested for anti-ACZ885 antibodies at the end of the study.
Arm/Group Title Canakinumab (ACZ885)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 156
Measure Type: Number
Unit of Measure: participants
0
4.Secondary Outcome
Title Pharmacokinetics
Hide Description Mean Clearance from serum in Liter per Day (CLD) in adult participants >=18, pediatric participants <18 with body weight >40 kg and pediatric participants <18 with body weight <=40 kg.
Time Frame Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population defined as all participants who received at least 1 dose of study drug. 3 participants were excluded because dosing information was not available at the time of analysis.
Arm/Group Title Canakinumab (ACZ885)
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 163
Mean (Standard Deviation)
Unit of Measure: L/day
Adult Participants >= 18 years 0.179  (0.084)
Pediatric Participants <18 years and >40 kg 0.180  (0.145)
Pediatric Participants <18 years and <= 40 kg 0.083  (0.033)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Canakinumab (ACZ885)
Hide Arm/Group Description 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.
All-Cause Mortality
Canakinumab (ACZ885)
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Canakinumab (ACZ885)
Affected / at Risk (%)
Total   18/166 (10.84%) 
Congenital, familial and genetic disorders   
Muckle-Wells syndrome  1  1/166 (0.60%) 
Gastrointestinal disorders   
Radicular cyst  1  1/166 (0.60%) 
Infections and infestations   
Abdominal abscess  1  1/166 (0.60%) 
Appendicitis  1  1/166 (0.60%) 
Bronchitis  1  1/166 (0.60%) 
Cellulitis  1  1/166 (0.60%) 
Chronic tonsillitis  1  1/166 (0.60%) 
H1N1 influenza  1  1/166 (0.60%) 
Pneumonia  1  2/166 (1.20%) 
Tonsillitis  1  2/166 (1.20%) 
Injury, poisoning and procedural complications   
Drug exposure during pregnancy  1  1/166 (0.60%) 
Forearm fracture  1  1/166 (0.60%) 
Investigations   
Hepatitis C antibody positive  1  1/166 (0.60%) 
Transaminases increased  1  1/166 (0.60%) 
Nervous system disorders   
Carpal tunnel syndrome  1  1/166 (0.60%) 
Headache  1  1/166 (0.60%) 
Nerve root compression  1  1/166 (0.60%) 
Pregnancy, puerperium and perinatal conditions   
Pregnancy  1  2/166 (1.20%) 
Psychiatric disorders   
Depression  1  1/166 (0.60%) 
Renal and urinary disorders   
Nephrotic syndrome  1  1/166 (0.60%) 
Social circumstances   
Miscarriage of partner  1  1/166 (0.60%) 
Surgical and medical procedures   
Abortion induced  1  2/166 (1.20%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Canakinumab (ACZ885)
Affected / at Risk (%)
Total   124/166 (74.70%) 
Gastrointestinal disorders   
Abdominal pain  1  13/166 (7.83%) 
Diarrhoea  1  18/166 (10.84%) 
Nausea  1  11/166 (6.63%) 
Vomiting  1  16/166 (9.64%) 
General disorders   
Pyrexia  1  18/166 (10.84%) 
Infections and infestations   
Bronchitis  1  18/166 (10.84%) 
Gastroenteritis  1  12/166 (7.23%) 
Nasopharyngitis  1  48/166 (28.92%) 
Rhinitis  1  27/166 (16.27%) 
Upper respiratory tract infection  1  17/166 (10.24%) 
Injury, poisoning and procedural complications   
Fall  1  11/166 (6.63%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  24/166 (14.46%) 
Back pain  1  12/166 (7.23%) 
Pain in extremity  1  9/166 (5.42%) 
Nervous system disorders   
Headache  1  33/166 (19.88%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  16/166 (9.64%) 
Oropharyngeal pain  1  14/166 (8.43%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
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 terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
Results Point of Contact
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
Responsible Party: Novartis
ClinicalTrials.gov Identifier: NCT00685373     History of Changes
Other Study ID Numbers: CACZ885D2306
First Submitted: May 27, 2008
First Posted: May 28, 2008
Results First Submitted: April 25, 2011
Results First Posted: May 27, 2011
Last Update Posted: November 4, 2016