Back to results

A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Still's Disease (SJIA and AOSD) (anaSTILLs)

Primary Purpose

Still's Disease, Adult-Onset, Still's Disease, Juvenile-Onset

Status

Terminated

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

anakinra

Placebo

About this trial

This is an interventional treatment trial for Still's Disease, Adult-Onset focused on measuring Interleukin 1 receptor antagonist, IL-1 receptor antagonist, Kineret, anakinra, Adult-Onset Still's Disease, Systemic Juvenile Idiopathic Arthritis

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

University of Alabama Birmingham
Attune Health
Rady Children's Hospital & Health Center
The Children's Hospital Colorado
University of Florida
University of Miami
Nicklaus Children's Hospital
University of Iowa Hospitals and Clinics
Children's Mercy Hospital and Clinics
University of Louisville School of Medicine Research Foundation
Beth Israel Deaconess Medical Center
Boston Children's Hospital
University of Michigan
Saint Paul Rheumatology
University of Minnesota
Saint Louis University
Hackensack University Medical Center
Institute for Rheumatic and Autoimmune Diseases
Hospital for Special Surgery
Columbia University Medical Center
UNC Hospitals
Duke Children's Hospital and Health Center
Wake Forest Baptist Brenner Medical Center
MetroHealth System
Nationwide Children's Hospital
Children's Hospital of Pittsburgh of UPMC
Monroe Carell Jr. Children's Hospital at Vanderbilt
Baylor Research Institute
Univ of TX Southwestern Medical Center Dallas - Texas Scottish Rite Hospital for Children
Texas Children's Hospital
University of Texas Health Science Center at San Antonio
University of Utah Hospitals and Clinics
University of Vermont Medical Center
Virginia Commonwealth University
University of Washington
Medical College of Wisconsin
University of Calgary - Alberta Children's Hospital
University of Calgary
The Hospital for Sick Children

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

anakinra

Placebo

Arm Description

2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day)

Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

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.

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.

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.

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.

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

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.

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.

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.

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.

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.

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.

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.

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.

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.

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

Absence of fever during the 24 hours preceding week 1.

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.

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.

Change From Baseline in CRP.

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

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.

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.

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.

Change From Baseline in CRP.

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

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

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

Change From Baseline in Platelet Count.

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

Change From Baseline in Ferritin.

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

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).

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.

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.

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

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

Percentage Decrease of the Glucocorticoid Dose From Baseline.

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

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.

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.

Proportion of Patients With Macrophage Activation Syndrome (MAS).

Proportion of patients with Macrophage Activation Syndrome (MAS).

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

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

Proportion of Patients With Neutralizing Antibodies.

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

Anakinra Serum Pre-dose Concentrations.

Week 2 reported here.

Anakinra Serum Pharmacokinetic Parameters: Cmax,

PK parameters only available for 2 patients.

Anakinra Serum Pharmacokinetic Parameters, Tmax and T½

PK parameters only available for 2 patients

Anakinra Serum Pharmacokinetic Parameter: AUC 0-24 h

PK parameters only available for 2 patients

Anakinra Serum Pharmacokinetic Parameter: CL/F

Pharmacokinetic parameters only available for 2 patients

Anakinra Serum Pharmacokinetic Parameter: Vd/F

PK parameters only available for 2 patients

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.

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.

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.

Change From Baseline in IL-6.

Only results from Week 2 reported here.

Change From Baseline in IL-18.

Only results from Week 2 reported here

Change From Baseline in Serum Calprotectin.

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

Change From Baseline in Neopterin.

Only results from Week 2 reported here

Full Information

NCT ID

NCT03265132

First Posted

August 25, 2017

Last Updated

June 9, 2021

Sponsor

Swedish Orphan Biovitrum

1. Study Identification

Unique Protocol Identification Number

NCT03265132

Brief Title

A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Still's Disease (SJIA and AOSD)

Acronym

anaSTILLs

Official Title

A Randomized, Double-blind, Placebo-controlled, Multicenter, Phase 3 Efficacy and Safety Study of 2 Dose Levels of Subcutaneous Anakinra (Kineret®) in Patients With Still's Disease (SJIA and AOSD)

Study Type

Interventional

2. Study Status

Record Verification Date

June 2021

Overall Recruitment Status

Terminated

Why Stopped

Meeting enrolment target (81 pat) will not be feasible within reasonable time.

Study Start Date

September 26, 2017 (Actual)

Primary Completion Date

February 13, 2019 (Actual)

Study Completion Date

May 23, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Swedish Orphan Biovitrum

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The aim of this study is to demonstrate the efficacy and to evaluate the safety, pharmacokinetics (PK) and immunogenicity of anakinra in patients with newly diagnosed Still's disease, including SJIA (Systemic juvenile idiopathic arthritis) and AOSD (Adult-onset Still's disease).

Detailed Description

The study consists of a 12-week, randomized, double-blind, placebo controlled period with two dose levels of anakinra and a 4-week safety follow-up after last dose of investigational medicinal product (IMP). The primary endpoint will be evaluated at Week 2. Sustained efficacy and time to study drug discontinuation will be evaluated during the full study period. A screening visit is optional and may be done to identify patients that could be suitable for the study. During the study 6 visits and 2 telephone contacts are scheduled i.e., Day 1 (baseline visit), Day 4Tel, Week 1, Week 2, Week 4, Week 8, Week 12 and Week 16Tel (End of Study). Patients will be randomly assigned to study drug, after they meet all of the inclusion criteria and none of the exclusion criteria. Patients will receive treatment for 12 weeks, either anakinra or placebo. Patients will be randomized to anakinra in a dose of either 2 or 4 mg/kg/day, with a maximum dose of 100 or 200 mg once daily, respectively. Patients will be randomized to placebo with corresponding volumes for each of the two anakinra dose levels.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Still's Disease, Adult-Onset, Still's Disease, Juvenile-Onset

Keywords

Interleukin 1 receptor antagonist, IL-1 receptor antagonist, Kineret, anakinra, Adult-Onset Still's Disease, Systemic Juvenile Idiopathic Arthritis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

13 (Actual)

8. Arms, Groups, and Interventions

Arm Title

anakinra

Arm Type

Experimental

Arm Description

2 mg/kg/day (max 100 mg/day) or 4 mg/kg/day (max 200 mg/day)

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Corresponding volume to anakinra 2 mg/kg/day or 4 mg/kg/day

Intervention Type

Biological

Intervention Name(s)

anakinra

Other Intervention Name(s)

Kineret

Intervention Description

sub cutaneous injection

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

sub cutaneous injection

Primary Outcome Measure Information:

Title

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

Description

Time Frame

Week 2

Secondary Outcome Measure Information:

Title

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

Description

Time Frame

Week 1

Title

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

Description

Time Frame

Week 1

Title

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

Description

Time Frame

Week 1

Title

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

Description

Time Frame

Week 1

Title

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

Description

Time Frame

Week 2

Title

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

Description

Time Frame

Week 2

Title

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

Description

Time Frame

Week 2

Title

Proportion of Responders in Physician Global Assessment of Disease Activity.

Description

Time Frame

Week 2

Title

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

Description

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.

Time Frame

Week 2

Title

Proportion of Responders in Number of Joints With Active Arthritis.

Description

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

Time Frame

Week 2

Title

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

Description

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

Time Frame

Week 2

Title

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

Description

Time Frame

Week 2

Title

Proportion of Responders in CRP (mg/L).

Description

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

Time Frame

Week 2

Title

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

Description

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

Time Frame

Week 2

Title

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

Description

Absence of fever during the 24 hours preceding week 1.

Time Frame

Week 1

Title

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

Description

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

Time Frame

Day 1 and Week 1

Title

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

Description

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.

Time Frame

Day 1 and Week 1

Title

Change From Baseline in CRP.

Description

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

Time Frame

Day 1 and Week 1

Title

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

Description

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.

Time Frame

Week 12

Title

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

Description

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

Time Frame

Week 2, Week 4, Week 8 and Week 12

Title

Proportion of Patients With Absence of Rash.

Description

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.

Time Frame

Week 2

Title

Change From Baseline in CRP.

Description

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

Time Frame

Week 2

Title

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

Description

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

Time Frame

Week 2

Title

Change From Baseline in Platelet Count.

Description

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

Time Frame

Week 2

Title

Change From Baseline in Ferritin.

Description

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

Time Frame

Week 2

Title

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

Description

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

Time Frame

Week 2

Title

Time to Study Drug Discontinuation for Any Reason.

Description

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.

Time Frame

From Day 1 to Week12

Title

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

Description

Time Frame

From Day 1 to Week12

Title

Proportion of Patients Who Have Initiated Tapering of Glucocorticoids.

Description

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

Time Frame

From Week 2 to Week12

Title

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

Description

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

Time Frame

From Week 2 to Week12

Title

Percentage Decrease of the Glucocorticoid Dose From Baseline.

Description

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

Time Frame

From Day 1 to Week12

Title

Proportion of Patients With at Least One Adverse Event.

Description

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

Time Frame

From Day 1 to Week 16

Title

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

Description

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

Time Frame

From Informed consent to Week 16

Title

Proportion of Patients With Macrophage Activation Syndrome (MAS).

Description

Proportion of patients with Macrophage Activation Syndrome (MAS).

Time Frame

From Day 1 to Week 16

Title

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

Description

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

Time Frame

Week 2

Title

Proportion of Patients With Neutralizing Antibodies.

Description

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

Time Frame

Week 2

Title

Anakinra Serum Pre-dose Concentrations.

Description

Week 2 reported here.

Time Frame

Week 2

Title

Anakinra Serum Pharmacokinetic Parameters: Cmax,

Description

PK parameters only available for 2 patients.

Time Frame

Week 12

Title

Anakinra Serum Pharmacokinetic Parameters, Tmax and T½

Description

PK parameters only available for 2 patients

Time Frame

Week 12

Title

Anakinra Serum Pharmacokinetic Parameter: AUC 0-24 h

Description

PK parameters only available for 2 patients

Time Frame

Week 12

Title

Anakinra Serum Pharmacokinetic Parameter: CL/F

Description

Pharmacokinetic parameters only available for 2 patients

Time Frame

Week 12

Title

Anakinra Serum Pharmacokinetic Parameter: Vd/F

Description

PK parameters only available for 2 patients

Time Frame

Week 12

Title

Change From Baseline in JADAS27.

Description

Time Frame

Week 2

Title

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

Description

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

Time Frame

Week 2

Title

Proportion of Patients With Inactive Disease.

Description

Time Frame

Week 12

Title

Change From Baseline in IL-6.

Description

Only results from Week 2 reported here.

Time Frame

Week 2

Title

Change From Baseline in IL-18.

Description

Only results from Week 2 reported here

Time Frame

Week 2

Title

Change From Baseline in Serum Calprotectin.

Description

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

Time Frame

Week 2

Title

Change From Baseline in Neopterin.

Description

Only results from Week 2 reported here

Time Frame

Week 2

10. Eligibility

Sex

All

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Sven Ohlman, MD PhD

Organizational Affiliation

Swedish Orphan Biovitrum

Official's Role

Study Director

Facility Information:

Facility Name

University of Alabama Birmingham

City

Birmingham

State/Province

Alabama

ZIP/Postal Code

35294

Country

United States

Facility Name

Attune Health

City

Beverly Hills

State/Province

California

ZIP/Postal Code

90211

Country

United States

Facility Name

Rady Children's Hospital & Health Center

City

San Diego

State/Province

California

ZIP/Postal Code

92123

Country

United States

Facility Name

The Children's Hospital Colorado

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

University of Florida

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32611

Country

United States

Facility Name

University of Miami

City

Miami

State/Province

Florida

ZIP/Postal Code

33124

Country

United States

Facility Name

Nicklaus Children's Hospital

City

Miami

State/Province

Florida

ZIP/Postal Code

33155

Country

United States

Facility Name

University of Iowa Hospitals and Clinics

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Facility Name

Children's Mercy Hospital and Clinics

City

Kansas City

State/Province

Kansas

ZIP/Postal Code

67208

Country

United States

Facility Name

University of Louisville School of Medicine Research Foundation

City

Louisville

State/Province

Kentucky

ZIP/Postal Code

40202

Country

United States

Facility Name

Beth Israel Deaconess Medical Center

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Boston Children's Hospital

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

University of Michigan

City

Ann Arbor

State/Province

Michigan

ZIP/Postal Code

48109

Country

United States

Facility Name

Saint Paul Rheumatology

City

Eagan

State/Province

Minnesota

ZIP/Postal Code

55121

Country

United States

Facility Name

University of Minnesota

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55454

Country

United States

Facility Name

Saint Louis University

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63104

Country

United States

Facility Name

Hackensack University Medical Center

City

Hackensack

State/Province

New Jersey

ZIP/Postal Code

07601

Country

United States

Facility Name

Institute for Rheumatic and Autoimmune Diseases

City

Summit

State/Province

New Jersey

ZIP/Postal Code

07901

Country

United States

Facility Name

Hospital for Special Surgery

City

New York

State/Province

New York

ZIP/Postal Code

10021

Country

United States

Facility Name

Columbia University Medical Center

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

Facility Name

UNC Hospitals

City

Chapel Hill

State/Province

North Carolina

ZIP/Postal Code

27514

Country

United States

Facility Name

Duke Children's Hospital and Health Center

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27710

Country

United States

Facility Name

Wake Forest Baptist Brenner Medical Center

City

Winston-Salem

State/Province

North Carolina

ZIP/Postal Code

27157

Country

United States

Facility Name

MetroHealth System

City

Cleveland

State/Province

Ohio

ZIP/Postal Code

44109

Country

United States

Facility Name

Nationwide Children's Hospital

City

Columbus

State/Province

Ohio

ZIP/Postal Code

43205

Country

United States

Facility Name

Children's Hospital of Pittsburgh of UPMC

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15224

Country

United States

Facility Name

Monroe Carell Jr. Children's Hospital at Vanderbilt

City

Nashville

State/Province

Tennessee

ZIP/Postal Code

37232

Country

United States

Facility Name

Baylor Research Institute

City

Dallas

State/Province

Texas

ZIP/Postal Code

75204

Country

United States

Facility Name

Univ of TX Southwestern Medical Center Dallas - Texas Scottish Rite Hospital for Children

City

Dallas

State/Province

Texas

ZIP/Postal Code

75219

Country

United States

Facility Name

Texas Children's Hospital

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Name

University of Texas Health Science Center at San Antonio

City

San Antonio

State/Province

Texas

ZIP/Postal Code

78229

Country

United States

Facility Name

University of Utah Hospitals and Clinics

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84113

Country

United States

Facility Name

University of Vermont Medical Center

City

Burlington

State/Province

Vermont

ZIP/Postal Code

05401

Country

United States

Facility Name

Virginia Commonwealth University

City

Richmond

State/Province

Virginia

ZIP/Postal Code

23298

Country

United States

Facility Name

University of Washington

City

Seattle

State/Province

Washington

ZIP/Postal Code

98195

Country

United States

Facility Name

Medical College of Wisconsin

City

Milwaukee

State/Province

Wisconsin

ZIP/Postal Code

53226

Country

United States

Facility Name

University of Calgary - Alberta Children's Hospital

City

Calgary

Country

Canada

Facility Name

University of Calgary

City

Calgary

Country

Canada

Facility Name

The Hospital for Sick Children

City

Toronto

Country

Canada

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

A Study to Evaluate Efficacy and Safety of Anakinra in the Treatment of Still's Disease (SJIA and AOSD)

We'll reach out to this number within 24 hrs