A Study to Evaluate Efficacy and Safety of Anakinra in th... | NCT03265132

Status Module

Description Module

Label	Type	Description	Intervention Names
anakinra	Experimental	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day)	Biological: anakinra
Placebo	Placebo Comparator	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day	Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
anakinra	Biological	sub cutaneous injection	anakinra	Kineret

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Proportion of ACR30 Responders With Absence of Fever Attributable to the Disease During the 7 Days Preceding Week 2.	ACR30 response is defined as an improvement of ≥ 30% from baseline in at least 3 of any 6 variables listed below. Also no more than 1 of the 6 variables may worsen by >30% from baseline. (ACR: American College of Rheumatology) Physician global assessment of disease activity - Assessed on a Visual Analogue Scale (VAS) from no disease activity (0 mm) to very severe disease activity (100 mm). Patient/parent global assessment of overall well-being - Assessed on a VAS from very well (0 mm) to very poor (100 mm). Number of joints with active arthritis. Number of joints with limitation of motion. Assessment of physical function - Patient Reported Outcome instruments : Childhood Health Assessment Questionnaire (CHAQ) /Stanford Health Assessment Questionnaire (SHAQ). C-Reactive Protein (CRP) (mg/L).	Week 2

Secondary Outcomes

Measure	Description	Time Frame
Proportion of ACR30 Responders With Absence of Fever During 24 Hours Preceding Week 1.	ACR30 response is defined as an improvement of ≥ 30% from baseline in at least 3 of any 6 variables listed in the description of the primary outcome measure. Also no more than 1 of the 6 variables may worsen by >30% from baseline.	Week 1

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Signed informed consent.
Male and female patients with a body weight ≥ 10 kg.
Diagnosis of Still's disease.
If currently on glucocorticoid treatment, a stable dose for at least 1 week prior to randomization.
If currently on methotrexate treatment, a stable dose for at least 8 weeks prior to randomization.
Active disease.
Female patients of childbearing potential must use an effective method of contraception during the study (abstinence being a possible option) as well as present a negative pregnancy test prior to randomization.
Negative interferon-gamma release assay or Purified protein derivative ( PPD) test within 2 months prior to randomization. If not available, a test should be performed at day of randomization.

Exclusion Criteria:

Diagnosis of Still's disease more than 6 months prior to randomization.
Previous randomization into this study.
Participation in another concurrent clinical interventional study within 30 days of randomization.
Treatment with an investigational drug within 5 half-lives prior to randomization.
Previous or current treatment with anakinra, canakinumab or any other IL-1 inhibitor.
Use of the following therapies prior to randomization:
- Narcotic analgesics within 24 hours prior to randomization.
- Dapsone or etanercept within 3 weeks prior to randomization.
- Intraarticular, intramuscular or intravenous administration of glucocorticoids or intravenous immunoglobulin (Ig) within 4 weeks prior to randomization.
- Intravenous Ig with proven Still's disease modifying effect, leflunomide, infliximab or adalimumab within 8 weeks prior to randomization.
- Thalidomide, cyclosporine, mycophenolate mofetil, 6-mercaptopurine, azathioprine, cyclophosphamide, chlorambucil or any other immunosuppressant within 12 weeks prior to randomization.
- Tocilizumab within 12 weeks prior to randomization or any other immunomodulatory medication within 4 half-lives prior to randomization
- Rituximab within 26 weeks prior to randomization.
Live vaccines within 1 month prior to randomization.
Known presence or suspicion of active, chronic or recurrent bacterial, fungal or viral infections, including tuberculosis, HIV infection or hepatitis B or C infection.
Clinical evidence of liver disease or liver injury.
Presence of severe renal function impairment.
Presence of neutropenia.
Presence or suspicion of MAS at baseline.
A diagnosis of MAS within the last 2 months prior to randomization.
History of malignancy within 5 years.
Known hypersensitivity to E coli-derived proteins, or any components of Kineret® (anakinra).
Pregnant or lactating women.
Foreseeable inability to cooperate with given instructions or study procedures.
Presence of any medical or psychological condition or laboratory result that in the opinion of the investigator can interfere with the patient's ability to comply with the protocol requirements or makes the patient not appropriate for inclusion to the study and treatment with IMP.

Name	Affiliation	Role
Sven Ohlman, MD PhD	Swedish Orphan Biovitrum	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
University of Alabama Birmingham		Birmingham	Alabama	35294	United States
Attune Health

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
FG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Jul 3, 2018	Feb 12, 2020

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Proportion of ACR30 Responders With Absence of Fever Attributable to the Disease During the 7 Days Preceding Week 2.

ACR30 response is defined as an improvement of ≥ 30% from baseline in at least 3 of any 6 variables listed below. Also no more than 1 of the 6 variables may worsen by >30% from baseline. (ACR: American College of Rheumatology)

Physician global assessment of disease activity - Assessed on a Visual Analogue Scale (VAS) from no disease activity (0 mm) to very severe disease activity (100 mm).
Patient/parent global assessment of overall well-being - Assessed on a VAS from very well (0 mm) to very poor (100 mm).
Number of joints with active arthritis.
Number of joints with limitation of motion.
Assessment of physical function - Patient Reported Outcome instruments : Childhood Health Assessment Questionnaire (CHAQ) /Stanford Health Assessment Questionnaire (SHAQ).
C-Reactive Protein (CRP) (mg/L).

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG0006 OG0015

Title

Denominators

Categories

Title	Measurements
	OG0006 OG0010

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG001		Fisher Exact		0.0022		Risk Difference (RD)	1.0			2-Sided	95	0.42	1.00					Superiority

Secondary

Proportion of ACR30 Responders With Absence of Fever During 24 Hours Preceding Week 1.

ACR30 response is defined as an improvement of ≥ 30% from baseline in at least 3 of any 6 variables listed in the description of the primary outcome measure. Also no more than 1 of the 6 variables may worsen by >30% from baseline.

Posted

Count of Participants

Participants

Week 1

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of ACR50 Responders With Absence of Fever During 24 Hours Preceding Week 1.

ACR50 response is defined as an improvement of ≥ 50% from baseline in at least 3 of any 6 variables listed in the description of the primary outcome measure. Also no more than 1 of the 6 variables may worsen by >30% from baseline.

Posted

Count of Participants

Participants

Week 1

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of ACR70 Responders With Absence of Fever During 24 Hours Preceding Week 1.

ACR70 response is defined as an improvement of ≥ 70% from baseline in at least 3 of any 6 variables listed in the description of the primary outcome measure. Also no more than 1 of the 6 variables may worsen by >30% from baseline.

Posted

Count of Participants

Participants

Week 1

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of ACR90 Responders With Absence of Fever During 24 Hours Preceding Week 1.

ACR90 response is defined as an improvement of ≥ 90% from baseline in at least 3 of any 6 variables listed in the description of the primary outcome measure. Also no more than 1 of the 6 variables may worsen by >30% from baseline.

Posted

Count of Participants

Participants

Week 1

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of ACR50 Responders With Absence of Fever During 7 Days Preceding Week 2.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of ACR70 Responders With Absence of Fever During 7 Days Preceding Week 2.

ACR70 response is defined as an improvement of ≥ 70% from baseline in at least 3 of any 6 variables listed in the description of the primary outcome. Also no more than 1 of the 6 variables may worsen by >30% from baseline.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of ACR90 Responders With Absence of Fever During 7 Days Preceding Week 2.

ACR90 response is defined as an improvement of ≥ 90% from baseline in at least 3 of any 6 variables listed in the description of the primary outcome . Also no more than 1 of the 6 variables may worsen by >30% from baseline.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Responders in Physician Global Assessment of Disease Activity.

Assessed on a VAS from no disease activity (0 mm) to very severe disease activity (100 mm). Response is defined as an improvement of ≥ 30%, 50%, 70% and 90% from baseline. Only improvement of ≥90% at Week 2 reported here.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Responders in Patient/Parent Global Assessment of Overall Well-being.

Assessed on a VAS from very well (0 mm) to very poor. (100 mm). Response is defined as an improvement of ≥ 30%, 50%, 70% and 90% from baseline.Only improvement of ≥90% at Week 2 reported here.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Responders in Number of Joints With Active Arthritis.

Response is defined as an improvement of ≥ 30%, 50%, 70% and 90% from baseline.Only improvement of ≥90% at Week 2 reported here.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Responders in Number of Joints With Limitation of Motion.

Response is defined as an improvement of ≥ 30%, 50%, 70% and 90% from baseline.Only improvement of ≥90% at Week 2 reported here.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Responders in Assessment of Physical Function (CHAQ/SHAQ).

Childhood Health Assessment Questionnaire (CHAQ) and Stanford Health Assessment Questionnaire (SHAQ) assess physical and functional status (see Clinical protocol section 6.5.4.1.5). Response is defined as an improvement of ≥ 30%, 50%, 70% and 90% from baseline. Only improvement of ≥90% at Week 2 reported here.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Responders in CRP (mg/L).

Response is defined as an improvement of ≥ 30%, 50%, 70% and 90% from baseline. Only improvement of ≥90% at Week 2 reported here.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With Absence of Fever During the 7 Days Preceding Week 2.

Proportion of patients with absence of fever during the 7 days preceding Week 2.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With Absence of Fever During the 24 Hours Preceding Week 1.

Absence of fever during the 24 hours preceding week 1.

Posted

Count of Participants

Participants

Week 1

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Physician Global Assessment of Disease Activity at Week 1.

Change from baseline in Physician global assessment of disease activity measured on a VAS 0 (very well)-100 (very poor) at Week 1.

Posted

Mean

Standard Deviation

Day 1 and Week 1

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Patient/Parent Global Assessment of Overall Well-being at Week 1.

Change from baseline in patient/parent global assessment of overall well-being measured on a VAS 0 (very well)-100 (very poor) at Week 1.

Posted

Mean

Standard Deviation

Day 1 and Week 1

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in CRP.

Change from baseline in C-Reactive Protein (CRP). CRP is measured in mg/L.

Posted

Mean

Standard Deviation

mg/L

Day 1 and Week 1

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With Sustained ACR30, ACR50, ACR70 and ACR90 Response.

Proportion of patients that still meet the corresponding week 2 response with absence of fever in the preceding 7 days. Only the strictest criteria, ACR90, is reported here.

Posted

Count of Participants

Participants

Week 12

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With Sustained ACR30, ACR50, ACR70 and ACR90 Response in Relation to Glucocorticoid Tapering.

Please note no patients were treated with any systemic glucocorticoids at randomization. Hence no results available.

No patients were treated with any systemic glucocorticoids at randomization

Posted

Week 2, Week 4, Week 8 and Week 12

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With Absence of Rash.

Absence of rash is evaluated 24 hours preceding Week 1 and 7 days preceding Week 2, Week 4, Week 8 and Week 12. Only data at Week 2 reported here.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in CRP.

Change from baseline in CRP. Results at Week 2 reported here.

Posted

Mean

Standard Deviation

mg/L

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Hemoglobin (Hb). Results at Week 2 Reported Here.

Change from baseline in Hemoglobin (Hb). Results at Week 2 reported here.

Posted

Mean

Standard Deviation

g/dL

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Platelet Count.

Change from baseline in platelet count. Results at Week 2 reported here.

Posted

Mean

Standard Deviation

10^9/L

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Ferritin.

Change from baseline in ferritin. Results at Week 2 reported here.

Posted

Mean

Standard Deviation

ug/L

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Patient/Parent Global Assessment of Disease Related Pain.

Assessed on a VAS from no pain (0 mm) to very severe pain (100 mm).

Patients with baseline and week 2 data analyzed.

Posted

Mean

Standard Deviation

score on a scale

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Time to Study Drug Discontinuation for Any Reason.

Time to study drug discontinuation was analyzed using Kaplan-Meier curves. Number of patients with premature study drug discontinuation for any reason is reported here.

Posted

Count of Participants

Participants

From Day 1 to Week12

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Time to Study Drug Discontinuation Due to Lack of Efficacy or Progressive Disease.

Proportion of study drug discontinuation due to lack of efficacy or progressive disease was analyzed using Kaplan-Meier curves. Number of patients discontinuing study drug due to lack of efficacy or progressive disease is reported here.

Posted

Count of Participants

Participants

From Day 1 to Week12

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients Who Have Initiated Tapering of Glucocorticoids.

Please note no patients were treated with any systemic glucocorticoids at randomization. Hence no results available

No patients were treated with any systemic glucocorticoids at randomization

Posted

From Week 2 to Week12

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients That Have Decreased the Glucocorticoid Dose With at Least 50% From Baseline.

Please note no patients were treated with any systemic glucocorticoids at randomization. Hence no results available

No patients were treated with any systemic glucocorticoids at randomization

Posted

From Week 2 to Week12

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Percentage Decrease of the Glucocorticoid Dose From Baseline.

Please note no patients were treated with any systemic glucocorticoids at randomization. Hence no results available

No patients were treated with any systemic glucocorticoids at randomization

Posted

From Day 1 to Week12

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With at Least One Adverse Event.

All adverse events collected from start of study treatment up to 28 days after stopping study treatment.

Posted

Count of Participants

Participants

From Day 1 to Week 16

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With at Least One Serious Adverse Event Including Death.

Serious adverse events (SAEs) will be collected from informed consent up to 28 days after stopping study treatment.

Posted

Count of Participants

Participants

From Informed consent to Week 16

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With Macrophage Activation Syndrome (MAS).

Proportion of patients with Macrophage Activation Syndrome (MAS).

Posted

Count of Participants

Participants

From Day 1 to Week 16

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With Antidrug Antibodies (ADA) Against Anakinra.

Proportion of patients with antidrug antibodies (ADA) against anakinra.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With Neutralizing Antibodies.

Confirmed ADA positive samples will be analyzed for the presence of neutralizing antibodies.

Posted

Count of Participants

Participants

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Anakinra Serum Pre-dose Concentrations.

Week 2 reported here.

No data available for the placebo group as they did not receive anakinra.

Posted

Mean

Standard Deviation

ng/mL

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Anakinra Serum Pharmacokinetic Parameters: Cmax,

PK parameters only available for 2 patients.

PK parameters only available for 2 anakinra treated patients.

Posted

Mean

Standard Deviation

ng/mL

Week 12

ID	Title	Description
OG000	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection
OG001	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Anakinra Serum Pharmacokinetic Parameters, Tmax and T½

PK parameters only available for 2 patients

Posted

Mean

Standard Deviation

hours

Week 12

ID	Title	Description
OG000	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection
OG001	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Anakinra Serum Pharmacokinetic Parameter: AUC 0-24 h

PK parameters only available for 2 patients

PK parameters only available for 2 anakinra treated patients

Posted

Mean

Standard Deviation

h*ng/mL

Week 12

ID	Title	Description
OG000	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection
OG001	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Anakinra Serum Pharmacokinetic Parameter: CL/F

Pharmacokinetic parameters only available for 2 patients

Pharmacokinetic parameters only available for 2 anakinra treated patients

Posted

Mean

Standard Deviation

mL/h*kg

Week 12

ID	Title	Description
OG000	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection
OG001	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Anakinra Serum Pharmacokinetic Parameter: Vd/F

PK parameters only available for 2 patients

PK parameters only available for 2 anakinra treated patients

Posted

Mean

Standard Deviation

mL/kg

Week 12

ID	Title	Description
OG000	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection
OG001	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in JADAS27.

Juvenile Arthritis Disease Activity Score (JADAS) includes 4 measures: physician global assessment of disease activity, patient or parent global assessment of overall well-being, 27 active joint count, and CRP. The JADAS27 includes the 27 joints. JADAS27 is calculated as the sum of its four components, physician global assessment of disease activity converted to cm from the VAS (0=no activity, 10=maximum activity); patient global assessment of well-being converted to cm from the VAS (0=very well, 10=very poor); active joint count (0-27); and CRP. Prior to calculation CRP is truncated to a 0 - 10 scale according to the following formula: (CRP (mg/l) -10)/10. Before calculation, CRP values <10 mg/l are converted to 10 and CRP values >110 mg/l are converted to 110. The JADAS27 tool yields a global score of 0-57.

Only results from Week 2 reported here.

Posted

Mean

Standard Deviation

score on a scale

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts

Secondary

Number of Days Off School or Work Due to Still's Disease.

Number of days off school or work due to Still's disease week 1-2.

Posted

Mean

Standard Deviation

Days

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Proportion of Patients With Inactive Disease.

Inactive disease is a composite of the following parameters: no joints with active arthritis, no fever, no rash, no serositis, no splenomegaly, no generalized lymphadenopathy attributable to Still's disease, CRP level within normal limits, physician's global assessment of disease activity score below 10 mm on a 100 mm VAS and a documented morning stiffness ≤15 minutes.

Posted

Count of Participants

Participants

Week 12

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in IL-6.

Only results from Week 2 reported here.

Posted

Mean

Standard Deviation

ng/L

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in IL-18.

Only results from Week 2 reported here

Posted

Mean

Standard Deviation

ng/L

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Serum Calprotectin.

Change from baseline in serum calprotectin. Only results from Week 2 reported here

Posted

Mean

Standard Deviation

mg/L

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Neopterin.

Only results from Week 2 reported here

Posted

Mean

Standard Deviation

nmol/L

Week 2

ID	Title	Description
OG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
OG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection

Units	Counts
Participants	OG000

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Upper respiratory infection	Infections and infestations	MedDRA (Unspecified)	Systematic Assessment	EG0003 events3 affected6 at risk EG0010 events0 affected6 at risk
Ear infection	Infections and infestations	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Urinary tract infection	Infections and infestations	MedDRA (Unspecified)	Systematic Assessment	EG0000 events0 affected6 at risk EG0011 events1 affected6 at risk
Viral upper respiratory tract infection	Infections and infestations	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Anxiety	Psychiatric disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Depression	Psychiatric disorders	MedDRA (Unspecified)	Systematic Assessment	EG0000 events0 affected6 at risk EG0011 events1 affected6 at risk
Dizziness	Nervous system disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0011 events1 affected6 at risk
Headache	Nervous system disorders	MedDRA (Unspecified)	Systematic Assessment	EG0000 events0 affected6 at risk EG0011 events1 affected6 at risk
Neuralgia	Nervous system disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA (Unspecified)	Systematic Assessment	EG0002 events2 affected6 at risk EG0010 events0 affected6 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Vomiting	Gastrointestinal disorders	MedDRA (Unspecified)	Systematic Assessment	EG0002 events2 affected6 at risk EG0010 events0 affected6 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Nausea	Gastrointestinal disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Retching	Gastrointestinal disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Alopecia	Skin and subcutaneous tissue disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Night sweats	Skin and subcutaneous tissue disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Injection site reaction	General disorders	MedDRA (Unspecified)	Systematic Assessment	EG0002 events2 affected6 at risk EG0011 events1 affected6 at risk
Injection site erythema	General disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0011 events1 affected6 at risk
Injection site pain	General disorders	MedDRA (Unspecified)	Systematic Assessment	EG0000 events0 affected6 at risk EG0011 events1 affected6 at risk
Injection site rash	General disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Malaise	General disorders	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Vitamin D decrease	Investigations	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Joint injury	Injury, poisoning and procedural complications	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk
Thermal burn	Injury, poisoning and procedural complications	MedDRA (Unspecified)	Systematic Assessment	EG0001 events1 affected6 at risk EG0010 events0 affected6 at risk

ID	Term
D016706	Still's Disease, Adult-Onset
D001171	Arthritis, Juvenile

ID	Term
D001172	Arthritis, Rheumatoid
D001168	Arthritis
D007592	Joint Diseases
D009140	Musculoskeletal Diseases
D012216	Rheumatic Diseases
D003240	Connective Tissue Diseases
D017437	Skin and Connective Tissue Diseases
D001327	Autoimmune Diseases
D007154	Immune System Diseases

ID	Term
D016207	Cytokines
D036341	Intercellular Signaling Peptides and Proteins
D010455	Peptides
D000602	Amino Acids, Peptides, and Proteins
D011506	Proteins
D001685	Biological Factors

ID	Title	Description
BG000	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection
BG001	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection
BG002	Total	Total of all reporting groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Anakinra	2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day) anakinra: sub cutaneous injection	0	6	0	6	6	6
EG001	Placebo	Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day Placebo: sub cutaneous injection	0	6	1	6	4	6