Periodic Fevers Syndrome
Conditions
Keywords
Canakinumab,, interleukin-1, Periodic Fevers Syndrome, TRAPS, HIDS, crFMF, auto-inflammatory diseases, extension study
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.
Interventions
BIOLOGICALCanakinumab (AIN457)
Canakinumab solution for subcutaneous injection in vial which contained 150mg/mL canakinumab in 1 mL solution
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Japan
Participant flow
Pre-assignment 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).
Participants by arm
| Arm | Count |
|---|---|
| TRAPS 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. | 2 |
| HIDS/MKD 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. | 1 |
| cfFMF 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. | 1 |
| Total | 4 |
Baseline characteristics
| Characteristic | TRAPS | HIDS/MKD | cfFMF | Total |
|---|---|---|---|---|
| Age, Continuous | 20.0 Years STANDARD_DEVIATION 2.83 | 14 Years | 20 Years | 18.5 Years STANDARD_DEVIATION 3.42 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants | 1 Participants | 1 Participants | 4 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Female | 0 Participants | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 2 Participants | 0 Participants | 1 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 2 | 0 / 1 | 0 / 1 |
| other Total, other adverse events | 1 / 2 | 1 / 1 | 0 / 1 |
| serious Total, serious adverse events | 0 / 2 | 0 / 1 | 0 / 1 |
Outcome results
Primary
Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths
Participants were monitored for safety throughout the study.
Time frame: Participants were followed for the duration until approval, an expected average of 3 months.
Population: Safety set: The safety set included all participants who were treated in this extension study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| TRAPS | Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths | Serious adverse events | 0 Participants |
| TRAPS | Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths | Non-serious adverse events | 1 Participants |
| TRAPS | Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths | Deaths | 0 Participants |
| HIDS/MKD | Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths | Serious adverse events | 0 Participants |
| HIDS/MKD | Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths | Non-serious adverse events | 1 Participants |
| HIDS/MKD | Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths | Deaths | 0 Participants |
| cfFMF | Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths | Non-serious adverse events | 0 Participants |
| cfFMF | Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths | Deaths | 0 Participants |
| cfFMF | Number of Participants With Non-serious Adverse Events, Serious Adverse Events and Deaths | Serious adverse events | 0 Participants |