An Extention Study of Safety of Canakinumab in Japanese Patients With Periodic Fever Syndromes

• Sponsor: Novartis Pharmaceuticals (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02911857
• Collaborator: (none)
• Enrollment: 4
• Locations: 3
• Arm: 1
• Actual Duration (Months): 3.8
• Patients Per Site: 1.3
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

The primary objective of this study was to evaluate safety and tolerability of ACZ885 in this extension study. This extension study offered the opportunity for participants who completed Epoch 4 of the preceding CACZ885N2301 (NCT02059291) study to continue to be treated with ACZ885 until approval in Japan of the drug in Periodic Fever Syndromes or until development of ACZ885 in Periodic Fever Syndromes was suspended.

Condition or Disease	Intervention/Treatment	Phase
Periodic Fevers Syndrome	Biological: Canakinumab (AIN457)	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 4 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: 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))
• Actual Study Start Date: Oct 3, 2016
• Actual Primary Completion Date: Jan 27, 2017
• Actual Study Completion Date: Jan 27, 2017

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Canakinumab (ACZ885) 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.	Biological: Canakinumab (AIN457); Canakinumab solution for subcutaneous injection in vial which contained 150mg/mL canakinumab in 1 mL solution

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths [Participants were followed for the duration until approval, an expected average of 3 months.]

   Participants were monitored for safety throughout the study.

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Completed Epoch 4 of the CACZ885N2301 study in Japan before the approval of canakinumab in Japan

• Written informed consent. Parent or legal guardian's written informed consent and child's assent, if appropriate, are required before any assessment is performed for patients < 20 years of age

Exclusion Criteria:

• Any conditions or significant medical problems in which the investigator judges the patient should not enter this extension study

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novartis Investigative Site	Fukuoka city	Fukuoka	Japan	812-8582
2	Novartis Investigative Site	Yokohama-city	Kanagawa	Japan	236-0004
3	Novartis Investigative Site	Kyoto-city	Kyoto	Japan	606-8507

Sponsors and Collaborators

• Novartis Pharmaceuticals

Investigators

None specified.

Study Documents (Full-Text)

• Study Protocol - Jun 3, 2016
• Statistical Analysis Plan - Sep 12, 2016

More Information

Publications

None provided.

• Responsible Party: Novartis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT02911857
• Other Study ID Numbers: CACZ885N2301E2
• First Posted: Sep 22, 2016
• Last Update Posted: Aug 20, 2018
• Last Verified: Nov 1, 2017

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Novartis Pharmaceuticals: Canakinumab,; interleukin-1; Periodic Fevers Syndrome; TRAPS; HIDS; crFMF; auto-inflammatory diseases; extension study
• Additional relevant MeSH terms: Familial Mediterranean Fever; Amyloidosis; Syndrome; Fever; Hyperthermia

Study Results

Participant Flow

Pre-assignment Detail	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).

Arm/Group Title	TRAPS	HIDS/MKD	cfFMF
Arm/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.	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.	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.
Period Title: Overall Study
STARTED	2	1	1
COMPLETED	2	1	1
NOT COMPLETED	0	0	0

Baseline Characteristics

Arm/Group Title	TRAPS	HIDS/MKD	cfFMF	Total
Arm/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.	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.	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.	Total of all reporting groups
Overall Participants	2	1	1	4
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	20.0 (2.83)	14 (NA)	20 (NA)	18.5 (3.42)
Sex: Female, Male (Count of Participants)
Female	0 0%	1 100%	0 0%	1 25%
Male	2 100%	0 0%	1 100%	3 75%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%	0 0%
Asian	2 100%	1 100%	1 100%	4 100%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%	0 0%
Black or African American	0 0%	0 0%	0 0%	0 0%
White	0 0%	0 0%	0 0%	0 0%
More than one race	0 0%	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%	0 0%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths
Description	Participants were monitored for safety throughout the study.
Time Frame	Participants were followed for the duration until approval, an expected average of 3 months.

Outcome Measure Data

Analysis Population Description

Safety set: The safety set included all participants who were treated in this extension study.

Arm/Group Title	TRAPS	HIDS/MKD	cfFMF
Arm/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.	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.	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.
Measure Participants	2	1	1
Non-serious adverse events	1 50%	1 100%	0 0%
Serious adverse events	0 0%	0 0%	0 0%
Deaths	0 0%	0 0%	0 0%

Adverse Events

Time Frame	3 months
Arm/Group Title	TRAPS		HIDS		cfFMF
Arm/Group Description	TRAPS		HIDS		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.
All Cause Mortality
	TRAPS		HIDS		cfFMF
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/2 (0%)		0/1 (0%)		0/1 (0%)
Serious Adverse Events
	TRAPS		HIDS		cfFMF
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/2 (0%)		0/1 (0%)		0/1 (0%)
Other (Not Including Serious) Adverse Events
	TRAPS		HIDS		cfFMF
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/2 (50%)		1/1 (100%)		0/1 (0%)
Infections and infestations
NASOPHARYNGITIS	1/2 (50%)		1/1 (100%)		0/1 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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 or disclosure of trial results in their entirety.