Hereditary Periodic Fevers, Systemic Juvenile Idiopathic Arthritis
Conditions
Keywords
Juvenile Rheumatoid arthritis (JRA) chronic, systemic inflammatory disorder, painful joints, inflammation of the synovial membrane, auto-immune rheumatoid disease, reactive rheumatoid arthritis, Systemic Juvenile Rheumatoid arthritis (SJRA), Hereditary Periodic Fevers, Hereditary periodic fever syndrome(HPFS), Familial Mediterranean fever syndrome(FMFS), Hyperimmunoglobulinemia D, Tumor necrosis factor (TNF) receptor-associated periodic syndrome (TRAPS), Muckle-Wells syndrome (MWS), Familial cold auto inflammatory syndrome
Brief summary
The objective of this extension protocol is to collect safety data (serious and non-serious adverse events) and to provide continuous canakinumab to patients in France who completed study CACZ885G2301E1(NCT00891046), CACZ885G2306 (NCT02296424) or CACZ885N2301 (NCT02059291) until a decision regarding reimbursement in France is effective for canakinumab (Ilaris®) in these indications.
Interventions
DRUGcanakinumab
canakinumab
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 20 Years
Healthy volunteers
No
Inclusion criteria
Criteria applicable for patients with Systemic Juvenil Idiopathic Arthritis SJIA): Patients who have completed the international studies CACZ885G2301E1 or CACZ885G2306 without any significant safety issue according to Investigator's opinion. Patients who have completed the international CACZ885G2306 study and who successfully withdrew canakinumab treatment per protocol but with a disease relapse after the end of study visit will be allowed to participate in CACZ885GFR01 study (whatever the time of relapse from the end of study visit), if the investigator states that there is an indication to resume canakinumab. Patients who have participated in the international CACZ885G2306 study but could not be randomized and then have continued canakinumab in part I until the end of the study at a dose of 4 mg/kg every 4 weeks may be switched to CACZ885GFR01 study if the investigator thinks that, in the interest of the patient, there is an indication to taper off canakinumab dose after a prolonged remission. Criteria applicable for patients with HPF (TRAPS, HIDS, crFMF): Patients who have completed the international CACZ885N2301 study without any significant safety issue according to Investigator's opinion. Criteria applicable for all patients: Parent's or legal guardian's written informed consent and child's assent, if appropriate, or patient's written informed consent for patients ≥ 18 years of age must be obtained before any study related activity or assessment is performed.
Exclusion criteria
* History of recurring infections * Hypersensitivity to the active substance or to any of the excipients other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events | every 4 weeks up to 1 year | The objective of this protocol was to collect additional safety data (serious and non serious AEs) and to provide continuous Ilaris® (canakinumab) treatment to patients in France who completed CACZ885G2301E1, CACZ885N2301 or CACZ885G2306 studies. |
| All-cause Mortality | uo to 1 year | Number of participants who died for any reason during the study |
Countries
France
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Canakinumab Patients will continue the same dose as their last dose administered in the study CACZ885G2301E1, CACZ885N2301 or CACZ885G2306. For all indications, the maximum canakinumab dose is 4 mg/kg or 300 mg for patients ≥ 40 kg. Ilaris® dosage may be adjusted (or interrupted) according to the clinical response and to investigators judgment. | 31 |
| Total | 31 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
| Overall Study | Inclusion in the CACZ8852306 study | 1 |
| Overall Study | Lack of Efficacy | 4 |
| Overall Study | Patient no longer required study drug | 2 |
Baseline characteristics
| Characteristic | Canakinumab |
|---|---|
| Age, Continuous | 9.7 years STANDARD_DEVIATION 4.07 |
| Race/Ethnicity, Customized Caucasian | 29 Participants |
| Race/Ethnicity, Customized Other | 2 Participants |
| Sex: Female, Male Female | 17 Participants |
| Sex: Female, Male Male | 14 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 31 |
| other Total, other adverse events | 28 / 31 |
| serious Total, serious adverse events | 7 / 31 |
Outcome results
Primary
All-cause Mortality
Number of participants who died for any reason during the study
Time frame: uo to 1 year
Population: Safety set
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Canakinumab | All-cause Mortality | 0 Participants |
Primary
Number of Participants With Adverse Events
The objective of this protocol was to collect additional safety data (serious and non serious AEs) and to provide continuous Ilaris® (canakinumab) treatment to patients in France who completed CACZ885G2301E1, CACZ885N2301 or CACZ885G2306 studies.
Time frame: every 4 weeks up to 1 year
Population: Safety set: Safety set consisted of all patients who received at least one dose of study drug and had at least one post-treatment safety assessment in the study.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Canakinumab | Number of Participants With Adverse Events | Serious adverse events | 7 Participants |
| Canakinumab | Number of Participants With Adverse Events | Treatment emergent adverse events | 29 Participants |