Trial Outcomes & Findings for Canakinumab in Patients With Active Hyper-IgD Syndrome (NCT NCT01303380)
NCT ID: NCT01303380
Last Updated: 2015-11-05
Results Overview
A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a C-reactive protein (CRP) value \> 10 mg/L. Flares during a historical period were defined as most recent 6-months in which the participant has not received treatment for their HIDS other than symptomatic treatment with NSAIDs and/or corticosteroids.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
9 participants
Primary outcome timeframe
Historical period, Month 6 (End of treatment period)
Results posted on
2015-11-05
Participant Flow
The study was conducted at 3 centres in Spain.
A total of 10 participants were screened, 9 of which entered in the treatment period of the study, one participant was considered to be a screening failure.
Participant milestones
| Measure |
Canakinumab
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new Hyper-IgD with periodic fever syndrome (HIDS) flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Overall Study
STARTED
|
9
|
|
Overall Study
COMPLETED
|
8
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Canakinumab
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new Hyper-IgD with periodic fever syndrome (HIDS) flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Overall Study
Lack of compliance to study procedures
|
1
|
Baseline Characteristics
Canakinumab in Patients With Active Hyper-IgD Syndrome
Baseline characteristics by cohort
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Age, Continuous
|
17.3 years
n=39 Participants
|
|
Age, Customized
Children (2--11 years)
|
3 participants
n=39 Participants
|
|
Age, Customized
Adolescents (12--17 years)
|
3 participants
n=39 Participants
|
|
Age, Customized
Adults (18--64 years)
|
3 participants
n=39 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=39 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=39 Participants
|
|
Region of Enrollment
Spain
|
9 participants
n=39 Participants
|
PRIMARY outcome
Timeframe: Historical period, Month 6 (End of treatment period)Population: The primary analysis was performed in the Full Analysis set (FAS) population defined as all participants who received at least one dose of study treatment and had at least one post baseline assessment.
A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a C-reactive protein (CRP) value \> 10 mg/L. Flares during a historical period were defined as most recent 6-months in which the participant has not received treatment for their HIDS other than symptomatic treatment with NSAIDs and/or corticosteroids.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Number of Flares Per Participant During Historical Period and Treatment Period
Historical period
|
5 Number of flares
Interval 3.0 to 12.0
|
|
Number of Flares Per Participant During Historical Period and Treatment Period
Month 6
|
0 Number of flares
Interval 0.0 to 2.0
|
SECONDARY outcome
Timeframe: Month 6 (End of treatment period), Month 36 (End of Long term treatment Period 2)Population: The analysis was performed in the FAS population.
A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value \> 10 mg/L.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Number of Flares Per Participant at During Treatment Period and 24 Month Extension Period
Treatment period
|
0 Number of flares
Interval 0.0 to 2.0
|
|
Number of Flares Per Participant at During Treatment Period and 24 Month Extension Period
Long term treatment period
|
0 Number of flares
Interval 0.0 to 1.0
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population.
A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value \> 10 mg/L.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Number of Participants Who Flared at Month 6, Month 24 and Month 36
End of Long term treatment Period 1
|
4 Number of participants
|
|
Number of Participants Who Flared at Month 6, Month 24 and Month 36
Treatment period
|
2 Number of participants
|
|
Number of Participants Who Flared at Month 6, Month 24 and Month 36
End of Long term treatment Period 2
|
2 Number of participants
|
SECONDARY outcome
Timeframe: Any flare event [Baseline up to Month 36 (End of long term treatment period 2)]Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group, respectively.
Physician global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5- point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Day 45, Absent (n=9)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Day 45, Minimal (n=9)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Day 45,Mild (n=9)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Day 45,Moderate (n=9)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Day 45,Severe (n=9)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 1, Absent (n=2)
|
2 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 1, Minimal (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 1, Mild (n=2)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 1, Moderated (n=2)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 1, Severe (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 2, Absent (n=2)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 2, Minimal (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 2, Mild (n=2)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 2, Moderate (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 2, Severe (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 3, Absent (n=2)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 3, Minimal (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 3, Mild (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 3, Moderate (n=2)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Flare/Unscheduled visit 3, Severe (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Treatment period, Absent (n=7)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Treatment period, Minimal (n=7)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Treatment period, Mild (n=7)
|
4 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Treatment period, Moderate (n=7)
|
3 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Treatment period, Severe (n=7)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Follow-up period, Absent (n=8)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Follow-up period, Minimal (n=8)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Follow-up period, Mild (n=8)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Follow-up period, Moderate (n=8)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
End of Follow-up period, Severe (n=8)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 1, Absent (n=3)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 1, Minimal (n=3)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 1, Mild (n=3)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long-term 12 M period, Flare 1, Moderate (n=3)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long-term 12 M period, Flare 1, Severe (n=3)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 2, Absent (n=1)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long-term 12 M period, Flare 2, Minimal (n=1)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long-term 12 M period, Flare 2, Mild (n=1)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 2, Moderate (n=1)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 2, Severe (n=1)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 3, Absent (n=1)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 3, Minimal (n=1)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 3, Mild (n=1)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 3, Moderate (n=1)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 1, Flare 3, Severe (n=1)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 2, Absent (n=2)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 2, Minimal (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 2, Mild (n=2)
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 2, Moderate (n=2)
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score
Long term period 2, Severe (n=2)
|
0 Number of participants
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population.
Participant's global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5-point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe. Same investigator assessed the same participant throughout the study to ensure consistency between assessments. Investigators reviewed every participant's diary at each visit after their own clinical assessment.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Treatment period, Absent
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Treatment period, Minimal
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Treatment period, Mild
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Treatment period, Moderate
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Treatment period, Severe
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Follow-up period, Absent
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Follow-up period, Minimal
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Follow-up period, Mild
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Follow-up period, Moderate
|
2 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Follow-up period, Severe
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 1, Absent
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 1, Minimal
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 1, Mild
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 1, Moderate
|
2 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 1, Severe
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 2, Absent
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 2, Minimal
|
1 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 2, Mild
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 2, Moderate
|
0 Number of participants
|
|
Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score
Long term period 2, Severe
|
0 Number of participants
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group, respectively.
Participants were assessed by participants/parent (participants aged 6-18 years) for control of signs and symptoms associated with HIDS based on 5-point scale: 0 = No control; 1 = Poor control; 2 = Somewhat control; 3 = Good control; and 4= Excellent control.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Treatment period, No control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Treatment period, Poor control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Treatment period, Somewhat control (n=9)
|
11.11 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Treatment period, Good control (n=9)
|
33.33 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Treatment period, Excellent control (n=9)
|
55.56 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Follow-up period, No control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Follow-up period, Poor control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Follow-up period, Somewhat control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Follow-up period, Good control (n=9)
|
33.33 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Follow-up period, Excellent control (n=9)
|
66.67 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 1, No control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 1, Poor control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 1, Somewhat control (n=8)
|
12.5 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 1, Good control (n=8)
|
25 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 1, Excellent control (n=8)
|
62.5 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 2, No control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 2, Poor control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 2, Somewhat control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 2, Good control (n=8)
|
12.5 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Participants Assessed Symptom Control
Long term period 2, Excellent control (n=8
|
87.5 Percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group, respectively.
Participants were assessed by physician for control of signs and symptoms associated with HIDS based on 5-point scale: 0 = No control; 1 = Poor control; 2 = Somewhat control; 3 = Good control; and 4= Excellent control.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Treatment period, No control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Treatment period, Poor control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Treatment period, Somewhat control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Treatment period, Good control (n=9)
|
44.44 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Treatment period, Excellent control (n=9)
|
55.56 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Follow-up period, No control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Follow-up period, Poor control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Follow-up period, Somewhat control (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Follow-up period, Good control (n=9)
|
33.33 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Follow-up period, Excellent control (n=9)
|
66.67 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 1, No control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 1, Poor control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 1, Somewhat control (n=8)
|
12.5 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 1, Good control (n=8)
|
25 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 1, Excellent control (n=8)
|
62.5 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 2, No control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 2, Poor control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 2, Somewhat control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 2, Good control (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants With Defined Grades of Physician Assessed Symptom Control
Long term period 2, Excellent control (n=8
|
100 Percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group, respectively.
Fever severity was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Treatment period, Absent (n=9)
|
88.89 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Treatment period, Minimal (n=9)
|
11.11 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Treatment period, Mild (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Treatment period, Moderate (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Treatment period, Severe (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Follow-up period, Absent (n=9)
|
100 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Follow-up period, Minimal (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Follow-up period, Mild (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Follow-up period, Moderate (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Follow-up period, Severe (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 1, Absent (n=8)
|
75 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 1, Minimal (n=8)
|
12.5 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 1, Mild (n=8)
|
12.5 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 1, Moderate (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 1, Severe (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 2, Absent (n=8)
|
100 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 2, Minimal (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 2, Mild (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 2, Moderate (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Fever as Assessed by Physician's Global Assessment
Long term period 2, Severe (n=8)
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group, respectively.
Apthus ulcers were assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Treatment period, Absent (n=9)
|
88.89 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Treatment period, Minimal (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Treatment period, Mild (n=9)
|
11.11 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Treatment period, Moderate (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Treatment period, Severe (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Follow-up period, Absent (n=9)
|
100 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Follow-up period, Minimal (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Follow-up period, Mild (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Follow-up period, Moderate (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Follow-up period, Severe (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 1, Absent (n=8)
|
100 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 1, Minimal (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 1, Mild (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 1, Moderate (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 1, Severe (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 2, Absent (n=8)
|
100 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 2, Minimal (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 2, Mild (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 2, Moderate (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Apthus Ulcers as Assessed by Physician's Global Assessment
Long term period 2, Severe (n=8)
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group, respectively.
Lymphadenopathy severity was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Treatment period, Absent (n=9)
|
88.89 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Treatment period, Minimal (n=9)
|
11.11 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Treatment period, Mild (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Treatment period, Moderate (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Treatment period, Severe (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Follow-up period, Absent (n=9)
|
77.78 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Follow-up period, Minimal (n=9)
|
22.22 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Follow-up period, Mild (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Follow-up period, Moderate (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Follow-up period, Severe (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 1, Absent (n=8)
|
87.5 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 1, Minimal (n=8)
|
12.5 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 1, Mild (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 1, Moderate (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 1, Severe (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 2, Absent (n=8)
|
100 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 2, Minimal (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 2, Mild (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 2, Moderate (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Lymphadenopathy as Assessed by Physician's Global Assessment
Long term period 2, Severe (n=8)
|
0 Percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group, respectively.
Abdominal pain was assessed by physician after each flare using a 5-point scale: 0 =Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3 = Moderate; 4 = Severe.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Follow-up period, Minimal (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Follow-up period, Mild (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Follow-up period, Moderate (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Follow-up period, Severe (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 1, Absent (n=8)
|
87.5 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 1, Minimal (n=8)
|
12.5 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 1, Mild (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 1, Moderate (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 1, Severe (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 2, Absent (n=8)
|
100 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 2, Minimal (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 2, Mild (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 2, Moderate (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Long term period 2, Severe (n=8)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Treatment period, Absent (n=9)
|
100 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Treatment period, Minimal (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Treatment period, Mild (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Treatment period, Moderate (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Treatment period, Severe (n=9)
|
0 Percentage of participants
|
|
Percentage of Participants Experiencing Abdominal Pain as Assessed by Physician's Global Assessment
Follow-up period, Absent (n=9)
|
100 Percentage of participants
|
SECONDARY outcome
Timeframe: Day 1 (Baseline), Day 28Population: The analysis was performed in the FAS population.
Time to resolution of the initial flare after first dose of canakinumab was determined.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Time to Resolution of the Initial Flare After First Canakinumab Treatment
|
3 Days
Interval 1.0 to 5.0
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group respectively.
The C-reactive Protein (CRP) and/or Serum amyloid A protein (SAA) were used as inflammatory markers. The normal range of CRP was 0-10 mg/L.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Change From Baseline in Inflammation Markers Over Time up to Month 24
SAA- Long term period 2, (n=2)
|
-686.7 milligram(s)/liter
Interval -747.7 to -625.7
|
|
Change From Baseline in Inflammation Markers Over Time up to Month 24
CRP- 6 month (Treatment period), (n=6)
|
-120.5 milligram(s)/liter
Interval -164.0 to -23.0
|
|
Change From Baseline in Inflammation Markers Over Time up to Month 24
CRP- 6 month (Follow-up period), (n=9)
|
-111 milligram(s)/liter
Interval -164.0 to -23.0
|
|
Change From Baseline in Inflammation Markers Over Time up to Month 24
CRP- Long term period 1, (n=8)
|
-113.55 milligram(s)/liter
Interval -159.5 to -49.9
|
|
Change From Baseline in Inflammation Markers Over Time up to Month 24
CRP- Long term period 2, (n=8)
|
-125.2 milligram(s)/liter
Interval -164.0 to -34.1
|
|
Change From Baseline in Inflammation Markers Over Time up to Month 24
SAA- 6 month (Treatment period), (n=1)
|
-624.2 milligram(s)/liter
Interval -624.2 to -23.0
|
|
Change From Baseline in Inflammation Markers Over Time up to Month 24
SAA- 6 month (Follow-up period), (n=2)
|
-685.95 milligram(s)/liter
Interval -748.2 to -623.7
|
|
Change From Baseline in Inflammation Markers Over Time up to Month 24
SAA- Long term period 1, (n=2)
|
-684.1 milligram(s)/liter
Interval -749.2 to -619.0
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group respectively. Here "Number of participants analyzed" signifies the participants assessed for HAQ during study.
Participants were assessed for health-related quality of life (HRQoL) based on Health Assessment Questionnaire (HAQ). HAQ was an eight 8 categories questionnaire representing all activities related to physical function. Each category has various sub-categories, which were rated by the participants on a 4- point difficulty scale: 0 = any difficulty; 1 = some difficulty; 2 = much difficulty; 3 = unable to do. The total score was the mean of the 8 scores, and ranged from 0 (no disability) to 3 (completely disabled).
Outcome measures
| Measure |
Canakinumab
n=6 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Health Assessment Questionnaire (HAQ) Global Score in Adults Over Time
Treatment period, (n=3)
|
0 Score on a scale
Interval 0.0 to 0.0
|
|
Health Assessment Questionnaire (HAQ) Global Score in Adults Over Time
Follow-up period, (n=4)
|
0 Score on a scale
Interval 0.0 to 0.0
|
|
Health Assessment Questionnaire (HAQ) Global Score in Adults Over Time
Long term period 1, (n=5)
|
0 Score on a scale
Interval 0.0 to 1.0
|
|
Health Assessment Questionnaire (HAQ) Global Score in Adults Over Time
Long term period 2, (n=4)
|
0 Score on a scale
Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group respectively. Here "Number of participants analyzed" signifies the participants assessed for CHAQ during study.
Participants or their parents (participants aged 6 to 17 years) were assessed for HRQoL based on Childhood Health Assessment Questionnaire (CHAQ). CHAQ was an eight domain questionnaire representing functional capacity and independence, evaluated for previous week. Each domain was rated on a 4-point difficulty scale: 0 = any difficulty; 1 = some difficulty; 2 = much difficulty; 3 = unable to do.The total score is the mean from the 8 scores, and ranges from 0 (no disability) to 3 (completely disabled).
Outcome measures
| Measure |
Canakinumab
n=6 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Childhood Health Assessment Questionnaire (CHAQ) Global Score in Children Over Time
End to treatment period (n= 2)
|
0 Score on a scale
Interval 0.0 to 0.0
|
|
Childhood Health Assessment Questionnaire (CHAQ) Global Score in Children Over Time
End to follow-up period (n=4)
|
0.1 Score on a scale
Interval 0.0 to 1.0
|
|
Childhood Health Assessment Questionnaire (CHAQ) Global Score in Children Over Time
Long term period 1, (n=2)
|
0.1 Score on a scale
Interval 0.0 to 1.0
|
|
Childhood Health Assessment Questionnaire (CHAQ) Global Score in Children Over Time
Long term period 2, (n=1)
|
0.1 Score on a scale
Interval 0.0 to 1.0
|
SECONDARY outcome
Timeframe: Day 1 up to Month 6 (End of follow up)Population: The analysis was performed in the FAS population.
Participants who experienced a new HIDS flare between baseline and Week 4 and received an escalated dose of 450 mg of canakinumab every 6 weeks thereafter starting at Week 6 were determined.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Percentage of Participants Who Received Dose Up-titration During 6-month Treatment Period
|
22.22 Percentage of participants
|
SECONDARY outcome
Timeframe: Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)Population: The analysis was performed in the FAS population. The 'n' signifies those participants evaluable for this measure at specified time points for each group respectively.
Flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value \> 10 mg/L. The change in post canakinumab treatment flare duration during the study were assessed as compared to historical period.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Duration of Flares Experienced During the Study
Long term period 1, (n=6)
|
3.5 Days
Interval 2.0 to 8.0
|
|
Duration of Flares Experienced During the Study
Treatment period, (n=3)
|
3 Days
Interval 2.0 to 4.0
|
|
Duration of Flares Experienced During the Study
Follow-up period, (n=7)
|
4 Days
Interval 2.0 to 10.0
|
|
Duration of Flares Experienced During the Study
Long term period 2, (n=2)
|
8.5 Days
Interval 6.0 to 11.0
|
SECONDARY outcome
Timeframe: Last dose of canakinumab treatment in follow-up period to end of follow-up period (Day 337)Population: The analysis was performed on the FAS population. Here "Number of participants analysed" signifies the participants assessed for time to flare after the last dose of canakinumab during follow-up period.
The median time to flare by the participants after administration of the last dose of canakinumab during the follow-up period was analysed using Kaplan-Meier method.
Outcome measures
| Measure |
Canakinumab
n=7 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Time to Flare After the Last Dose of Canakinumab During the Follow-up Period
|
110 days
Interval 62.0 to 196.0
|
SECONDARY outcome
Timeframe: Day 1 (Start of study treatment) up to Month 36 (End of study)Population: The analysis was performed on Safety Set (SAF) population defined as all participants who received at least one application of study treatment and had at least one post-baseline safety assessment.
Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalisation, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Adverse events
|
9 Number of participants
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Serious adverse events
|
4 Number of participants
|
SECONDARY outcome
Timeframe: Baseline up to Month 36 (End of study)Population: The analysis was performed on the FAS population.
Participants who experienced flares were treated with corticosteroids and NSAIDs as rescue medication.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Participants Who Received Rescue Treatment
|
11.11 Percentage of participants
|
SECONDARY outcome
Timeframe: Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose)Population: The analysis was performed on the FAS population.
Canakinumab concentrations in serum were assessed for evaluating pharmacokinetics (PK) of the drug.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Serum Concentration-time Profile of Canakinumab
Day 4
|
28.8 microgram(s)/milliliter
Standard Deviation 7.9
|
|
Serum Concentration-time Profile of Canakinumab
Day 15
|
26.9 microgram(s)/milliliter
Standard Deviation 8
|
|
Serum Concentration-time Profile of Canakinumab
Day 43
|
12.7 microgram(s)/milliliter
Standard Deviation 4.6
|
|
Serum Concentration-time Profile of Canakinumab
Day 85
|
19.4 microgram(s)/milliliter
Standard Deviation 9.6
|
|
Serum Concentration-time Profile of Canakinumab
Day 127
|
23.8 microgram(s)/milliliter
Standard Deviation 12.8
|
|
Serum Concentration-time Profile of Canakinumab
Day 169
|
24 microgram(s)/milliliter
Standard Deviation 13.4
|
|
Serum Concentration-time Profile of Canakinumab
Day 197
|
12.6 microgram(s)/milliliter
Standard Deviation 13.7
|
|
Serum Concentration-time Profile of Canakinumab
Day 225
|
11.5 microgram(s)/milliliter
Standard Deviation 7.9
|
|
Serum Concentration-time Profile of Canakinumab
Day 253
|
6.5 microgram(s)/milliliter
Standard Deviation 4.8
|
|
Serum Concentration-time Profile of Canakinumab
Day 281
|
1.6 microgram(s)/milliliter
Standard Deviation 1.6
|
|
Serum Concentration-time Profile of Canakinumab
Day 309
|
0.9 microgram(s)/milliliter
Standard Deviation 1
|
|
Serum Concentration-time Profile of Canakinumab
Day 337
|
32.6 microgram(s)/milliliter
Standard Deviation 15.9
|
SECONDARY outcome
Timeframe: Day 1 (Pre-dose), Day 4, Day 15, Day 43, Day 85, Day 127, Day 169 (End of treatment period), Day 197, Day 225, Day 253, Day 281, Day 309, and Day 337 (End of follow-up period) (Post-dose)Population: The analysis was performed on the FAS population.
Pharmacodynamics of canakinumab was assessed by total IL-1β (sum of free and bound canakinumab) concentration, determined in serum by means of sandwich ELISA assay with limit of detection at 0.1 picogram/millilitre.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 4
|
27.3 picogram(s)/milliliter
Standard Deviation 25.2
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 15
|
22.2 picogram(s)/milliliter
Standard Deviation 14.7
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 43
|
21.8 picogram(s)/milliliter
Standard Deviation 13.9
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 85
|
27.3 picogram(s)/milliliter
Standard Deviation 14
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 127
|
24.3 picogram(s)/milliliter
Standard Deviation 12.1
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 169
|
32.5 picogram(s)/milliliter
Standard Deviation 17.9
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 197
|
16.3 picogram(s)/milliliter
Standard Deviation 5.9
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 225
|
17 picogram(s)/milliliter
Standard Deviation 8.3
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 253
|
10.2 picogram(s)/milliliter
Standard Deviation 5.8
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 281
|
4.9 picogram(s)/milliliter
Standard Deviation 3.3
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 309
|
3.7 picogram(s)/milliliter
Standard Deviation 2
|
|
Serum Concentration of Total Interleukin-1β Antibody (IL-1β)
Day 337
|
49.1 picogram(s)/milliliter
Standard Deviation 60.2
|
SECONDARY outcome
Timeframe: Baseline up to Month 36 (End of study)Population: The analysis was performed on the FAS population.
Immunogenicity assessment included determination of anti-canakinumab (ACZ885) antibodies in serum samples using bridging ECLIA assay.
Outcome measures
| Measure |
Canakinumab
n=9 Participants
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Number of Participants Exhibiting Anti-canakinumab Antibodies at Any Visit
|
0 Number of participants
|
Adverse Events
Canakinumab
Serious events: 4 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Canakinumab
n=9 participants at risk
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
General disorders
Disease progression
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Cellulitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Peritonitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Pneumonia
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Streptococcal bacteraemia
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Metabolism and nutrition disorders
Fluid overload
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Musculoskeletal and connective tissue disorders
Temporomandibular joint syndrome
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Nervous system disorders
Headache
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Skin and subcutaneous tissue disorders
Hidradenitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Vascular disorders
Hypertensive crisis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
Other adverse events
| Measure |
Canakinumab
n=9 participants at risk
Participants received body weight stratified dosage of canakinumab (4 mg/kg for participants less than or equal to (≤) 40 kg or 300 mg for participants more than (\>) 40 kg) as starting dose s.c. injection every 6 weeks during 6 months of treatment. The dose was escalated to additional 150 mg (2 mg/kg for participants ≤40 kg) dose at the moment of flare, and 450 mg (6 mg/kg for participants ≤40 kg) every 6 weeks, thereafter starting at Week 6 in participants who experienced a new HIDS flare between baseline and Week 4 as per investigator's discretion. If the flare occurred between Weeks 5-6, the participants received rescue medication and waited up to Week 6 to receive a total of 450 mg of canakinumab.
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Back pain
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
22.2%
2/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bone neoplasm
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Nervous system disorders
Dizziness
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Nervous system disorders
Headache
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Reproductive system and breast disorders
Vaginal ulceration
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Skin and subcutaneous tissue disorders
Erythema nodosum
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Skin and subcutaneous tissue disorders
Hidradenitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Surgical and medical procedures
Tooth extraction
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Cardiac disorders
Tachycardia
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Eye disorders
Conjunctival hyperaemia
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Eye disorders
Conjunctivitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Gastrointestinal disorders
Abdominal pain
|
22.2%
2/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Gastrointestinal disorders
Aphthous stomatitis
|
22.2%
2/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Gastrointestinal disorders
Diarrhoea
|
22.2%
2/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Gastrointestinal disorders
Dyspepsia
|
22.2%
2/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Gastrointestinal disorders
Toothache
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Gastrointestinal disorders
Vomiting
|
22.2%
2/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
General disorders
Impaired healing
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
General disorders
Influenza like illness
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
General disorders
Pyrexia
|
44.4%
4/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Bronchitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Candidiasis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Cellulitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Folliculitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Impetigo
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Infection
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Influenza
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Nasopharyngitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Pharyngotonsillitis
|
33.3%
3/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Respiratory tract infection
|
22.2%
2/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Sinusitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Tonsillitis
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Tonsillitis streptococcal
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Tooth infection
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Upper respiratory tract infection
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Urinary tract infection
|
22.2%
2/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Infections and infestations
Vulvovaginal mycotic infection
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
11.1%
1/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
22.2%
2/9 • Serious Adverse Events are monitored from date of First Participant First Visit (FPFV) until Last Participant Last Visit (LPLV). All other adverse events are monitored from First Participant First Treatment until LPLV.
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862 -778 -8300
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER