Clinical Trial Results:
An extension study of CACZ885N2301 (NCT02059291), multi-center, open label study of canakinumab in Japanese patients with Periodic Fever Syndromes (Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS), Hyper Immunoglobulin D Syndrome ((also known as mevalonate kinase deficiency) (HIDS/MKD), or colchicine resistant/intolerant Familial Mediterranean Fever (crFMF)) Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/CtrdWeb/home.novfor complete trial results.

Trial information Top of page
Trial identification
Sponsor protocol code	CACZ885N2301E2
Additional study identifiers
ISRCTN number	-
US NCT number	NCT02911857
WHO universal trial number (UTN)	-
Sponsors
Sponsor organisation name	Novartis Pharma AG
Sponsor organisation address	CH-4002, Basel, Switzerland,
Public contact	Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
Scientific contact	Clinical Disclosure Office, Novartis Pharma AG, 41 613241111,
Paediatric regulatory details
Is trial part of an agreed paediatric investigation plan (PIP)	No
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?	No
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?	Yes
Results analysis stage
Analysis stage	Final
Date of interim/final analysis	27 Jan 2017
Is this the analysis of the primary completion data?	No
Global end of trial reached?	Yes
Global end of trial date	27 Jan 2017
Was the trial ended prematurely?	No
General information about the trial
Main objective of the trial	The primary objective of this study was to evaluate safety and tolerability of canakinumab in this extension study.
Protection of trial subjects	The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.
Background therapy	-
Evidence for comparator	-
Actual start date of recruitment	03 Oct 2016
Long term follow-up planned	No
Independent data monitoring committee (IDMC) involvement?	No
Population of trial subjects
Number of subjects enrolled per country
Country: Number of subjects enrolled	Japan: 4
Worldwide total number of subjects	4
EEA total number of subjects	0
Number of subjects enrolled per age group
In utero	0
Preterm newborn - gestational age < 37 wk	0
Newborns (0-27 days)	0
Infants and toddlers (28 days-23 months)	0
Children (2-11 years)	0
Adolescents (12-17 years)	1
Adults (18-64 years)	3
From 65 to 84 years	0
85 years and over	0

Trial information Top of page

Subject disposition Top of page
Recruitment
Recruitment details	-
Pre-assignment
Screening details	Participants with Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS), Hyper Immunoglobulin D Syndrome (also known as mevalonate kinase deficiency (HIDS/MKD), or colchicine resistant/intolerant Familial Mediterranean Fever (crFMF) received study drug based on the final dose regimen received in CACZ885N2301 (NCT02059291).
Period 1
Period 1 title	Overall Study (overall period)
Is this the baseline period?	Yes
Allocation method	Non-randomised - controlled
Blinding used	Not blinded
Arms
Are arms mutually exclusive	Yes
Arm title	TRAPS
Arm description	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Arm type	Experimental
Investigational medicinal product name	Canakinumab
Investigational medicinal product code	CACZ885
Other name
Pharmaceutical forms	Solution for injection
Routes of administration	Subcutaneous use
Dosage and administration details	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Arm title	HIDS/MKD
Arm description	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Arm type	Experimental
Investigational medicinal product name	Canakinumab
Investigational medicinal product code	CACZ885
Other name
Pharmaceutical forms	Solution for injection
Routes of administration	Subcutaneous use
Dosage and administration details	Participant continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Arm title	cfFMF
Arm description	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Arm type	Experimental
Investigational medicinal product name	Canakinumab
Investigational medicinal product code	CACZ885
Other name
Pharmaceutical forms	Solution for injection
Routes of administration	Subcutaneous use
Dosage and administration details	Participant continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Number of subjects in period 1 TRAPS HIDS/MKD cfFMF Started 2 1 1 Completed 2 1 1

Subject disposition Top of page

Baseline characteristics Top of page
Baseline characteristics reporting groups
Reporting group title	TRAPS
Reporting group description	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Reporting group title	HIDS/MKD
Reporting group description	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Reporting group title	cfFMF
Reporting group description	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Reporting group values TRAPS HIDS/MKD cfFMF Total Number of subjects 2 1 1 4 Age categorical Units: Subjects     In utero 0 0 0 0     Preterm newborn infants (gestational age < 37 wks) 0 0 0 0     Newborns (0-27 days) 0 0 0 0     Infants and toddlers (28 days-23 months) 0 0 0 0     Children (2-11 years) 0 0 0 0     Adolescents (12-17 years) 0 1 0 1     Adults (18-64 years) 2 0 1 3     From 65-84 years 0 0 0 0     85 years and over 0 0 0 0 Age Continuous Units: Years     arithmetic mean (standard deviation) 20 ( 2.83 ) 14 ( 9999 ) 20 ( 9999 ) - Gender, Male/Female Units: Subjects     Female 0 1 0 1     Male 2 0 1 3

Baseline characteristics Top of page

End points Top of page
End points reporting groups
Reporting group title	TRAPS
Reporting group description	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Reporting group title	HIDS/MKD
Reporting group description	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.
Reporting group title	cfFMF
Reporting group description	Participants continued the study drug based on the final dose and regimen administered at the end of the CACZ885N2301 study. All participants received 1 or 2 ACZ885 subcutaneous injections every 4 or 8 weeks.

End points Top of page

Primary: Number of participants with non-serious adverse events, serious adverse events and deaths Top of page
End point title	Number of participants with non-serious adverse events, serious adverse events and deaths [1]
End point description	Participants were monitored for safety throughout the study.
End point type	Primary
End point timeframe	Participants were followed for the duration until approval, an expected average of 3 months.
Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Statistical analysis does not apply to this end point.
End point values TRAPS HIDS/MKD cfFMF Number of subjects analysed 2 1 1 Units: Participants     Non-serious adverse events 1 1 0     Serious adverse events 0 0 0     Deaths 0 0 0
No statistical analyses for this end point

Primary: Number of participants with non-serious adverse events, serious adverse events and deaths Top of page

Adverse events Top of page
Adverse events information
Timeframe for reporting adverse events	Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit.
Assessment type	Systematic
Dictionary used for adverse event reporting
Dictionary name	MedDRA
Dictionary version	19.1
Reporting groups
Reporting group title	HIDS
Reporting group description	HIDS
Reporting group title	TRAPS
Reporting group description	TRAPS
Serious adverse events HIDS TRAPS Total subjects affected by serious adverse events      subjects affected / exposed 0 / 1 (0.00%) 0 / 2 (0.00%)      number of deaths (all causes) 0 0      number of deaths resulting from adverse events 0 0
Frequency threshold for reporting non-serious adverse events: 1%
Non-serious adverse events HIDS TRAPS Total subjects affected by non serious adverse events      subjects affected / exposed 1 / 1 (100.00%) 1 / 2 (50.00%) Infections and infestations NASOPHARYNGITIS      subjects affected / exposed 1 / 1 (100.00%) 1 / 2 (50.00%)      occurrences all number 1 2

Adverse events Top of page

More information Top of page
Substantial protocol amendments (globally)
Were there any global substantial amendments to the protocol? No
Interruptions (globally)
Were there any global interruptions to the trial? No
Limitations and caveats
Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
Due to EudraCT system limitations, which EMA is aware of, data using 999 as data points in this record are not an accurate representation of the clinical trial results. Please use https://www.novctrd.com/CtrdWeb/home.novfor complete trial results.

More information Top of page