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| Name | Class |
|---|---|
| CMIC Co, Ltd. Japan | INDUSTRY |
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A study to demonstrate efficacy and safety of anakinra in pediatric and adult Japanese patients with Still's disease (Systemic juvenile idiopathic arthritis [SJIA] and Adult-onset Still's disease [AOSD]).
The study consists of two phases:
• Core phase comprising 2 weeks double blind placebo-controlled treatment, 52 weeks open label treatment and 4 weeks safety follow up (only for patients not entering the extension phase).
At the Week 54 visit, patients who consent and are eligible to continue anakinra treatment, will enter the extension phase and continue open label anakinra treatment.
• Extension phase comprising up to 26 weeks open label treatment and 4 weeks safety follow up.
The primary endpoint will be evaluated at Week 2 visit.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Anakinra | Experimental | 100 mg/day or 2 mg/kg/day of subcutaneous anakinra for those with a body weight ≥50 kg or <50 kg, respectively. |
|
| Placebo | Placebo Comparator | Corresponding volume to 100 mg/day or 2 mg/kg/day |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Anakinra | Drug | sub cutaneous daily injection |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| An improvement of ≥ 30% from baseline in physician global assessment of disease activity (visual analogue scale [VAS]). | ACR30 response with absence of fever attributable to the disease during the 7 days | Week 2 |
| An improvement of ≥ 30% from baseline in patient/parent global assessment of overall well-being (VAS). | ACR30 response with absence of fever attributable to the disease during the 7 days | Week 2 |
| An improvement of ≥ 30% from baseline in number of joints with active arthritis. | ACR30 response with absence of fever attributable to the disease during the 7 days | Week 2 |
| An improvement of ≥ 30% from baseline in number of joints with limitation of motion. | ACR30 response with absence of fever attributable to the disease during the 7 days | Week 2 |
| An improvement of ≥ 30% from baseline in assessment of physical function: Child health assessment questionnaire (CHAQ)/Stanford health assessment questionnaire (SHAQ). | ACR30 response with absence of fever attributable to the disease during the 7 days | Week 2 |
| An improvement of ≥ 30% from baseline in C-reactive protein (CRP) (mg/L). | ACR30 response with absence of fever attributable to the disease during the 7 days | Week 2 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in CRP. | To demonstrate the efficacy of anakinra compared to placebo in reducing inflammation in Still's disease. | Week 2 |
| Change in ferritin. | To demonstrate the efficacy of anakinra compared to placebo in reducing inflammation in Still's disease. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Previous or current treatment with anakinra, or any other Interleukin-1 (IL-1) inhibitor except for canakinumab. Previous treatment with canakinumab is allowed if canakinumab was discontinued for reasons other than lack of efficacy and after a washout period of minimum 130 days. Patients who have discontinued canakinumab because of insufficient effect or refractory disease are not allowed to be enrolled in the study.
Use of the following therapies prior to enrollment.
Live vaccines within 4 weeks prior to enrollment.
Known presence or suspicion of active, chronic, or recurrent bacterial, fungal, or viral infections, including but not limited to tuberculosis, HIV infection, Covid-19 infection, or hepatitis B or C infection at baseline. Patients with acute or chronic HBV.
Clinical evidence of liver disease or liver injury as indicated by presence of abnormal liver tests.
Presence of severe chronic kidney disease (CKD) grades 4 and 5.
Presence of neutropenia (absolute neutrophil count [ANC] < 1.5 x 10^9/L).
Presence of thrombocytopenia (platelets count < 100 x 10^9/L).
Presence or suspicion of MAS at baseline.
History or diagnosis of MAS within the last 4 weeks prior to enrollment.
After completion of the study Core Phase, patients who consent and are eligible to continue anakinra treatment, can enter the extension phase .
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| Name | Affiliation | Role |
|---|---|---|
| Masaaki Mori, MD | St. Marianna University Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Fukushima Medical University Hospital | Fukushima | Fukushima | Japan | |||
| Hyogo Prefectural Kobe Children's Hospital |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Jun 23, 2026 |
| ID | Term |
|---|---|
| D001171 | Arthritis, Juvenile |
| D016706 | Still's Disease, Adult-Onset |
| ID | Term |
|---|---|
| D001168 | Arthritis |
| D007592 | Joint Diseases |
| D009140 | Musculoskeletal Diseases |
| D012216 | Rheumatic Diseases |
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| ID | Term |
|---|---|
| D053590 | Interleukin 1 Receptor Antagonist Protein |
| ID | Term |
|---|---|
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
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Double blind
| Placebo | Drug | sub cutaneous daily injection |
|
| Week 2 |
| Change in haemoglobin. | To demonstrate the efficacy of anakinra compared to placebo in reducing inflammation in Still's disease. | Week 2 |
| Change in platelets count. | To demonstrate the efficacy of anakinra compared to placebo in reducing inflammation in Still's disease. | Week 2 |
| Change in white blood cells count. | To demonstrate the efficacy of anakinra compared to placebo in reducing inflammation in Still's disease. | Week 2 |
| Absence of fever during the 24 hours preceding the evaluation visit at Week 1. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Absence of rash during the 24 hours preceding the evaluation visit at Week 1. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| ACR30 response with absence of fever during the 24 hours preceding the evaluation visit at Week 1. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| ACR50 response with absence of fever during the 24 hours preceding the evaluation visit at Week 1. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| ACR70 response with absence of fever during the 24 hours preceding the evaluation visit at Week 1. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in physician global assessment of disease activity, measured on a VAS from no pain (0 mm) to very severe (100 mm). | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in patient/parent global assessment of overall well-being, measured on a VAS from no pain (0 mm) to very severe (100 mm). | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in patient/parent global assessment of disease related pain, measured on a VAS from no pain (0 mm) to very severe (100 mm). | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in swelling joints count. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in tender joints count. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in CRP. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in ferritin. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in haemoglobin. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in platelets count. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in white blood cells count. | To demonstrate early onset of efficacy of anakinra compared to placebo in Still's disease. | Week 1 |
| Change in the number/proportion of patients reporting problems by dimension of EQ-5D-Y Proxy (4-7 years). | To evaluate and compare the health status between anakinra treated patients and patients treated with placebo. | Week 2 |
| Change in the number/proportion of patients reporting problems by dimension of EQ-5D-Y (8-15 years). | To evaluate and compare the health status between anakinra treated patients and patients treated with placebo. | Week 2 |
| Change in the number/proportion of patients reporting problems by dimension of EQ-5D-3L (≥ 16 years). | To evaluate and compare the health status between anakinra treated patients and patients treated with placebo. | Week 2 |
| Absence of fever during the 7 days preceding the visit. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Absence of rash during the 7 days preceding the visit. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| ACR30 response with absence of fever during the 7 days preceding the visit. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| ACR50 response with absence of fever during the 7 days preceding the visit. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| ACR70 response with absence of fever during the 7 days preceding the visit. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| ACR90 response with absence of fever during the 7 days preceding the visit. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in physician global assessment of disease activity (VAS). | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in patient/parent global assessment of overall well-being (VAS). | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in patient/parent global assessment of disease related pain (VAS). | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in swelling joints count. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in tender joints count. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in CRP. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in ferritin. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in haemoglobin. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in platelets count. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Change in white blood cells count. | To evaluate efficacy of anakinra in Still's disease. | Week 4 to Week 54 |
| Occurrence of inactive disease. | Proportion of patients who reach inactive disease.Inactive disease is defined as no joints with active arthritis, no fever, no rash, serositis, no hepatosplenomegaly, 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 min. | Week 8 to Week 54 |
| Change in the number/proportion of patients reporting problems by dimension of EQ-5D-Y Proxy (4-7 years). | To evaluate the health status in anakinra treated patients with Still's disease. | Week 4 to Week 54 |
| Change in the number/proportion of patients reporting problems by dimension of EQ-5D-Y (8-15 years). | To evaluate the health status in anakinra treated patients with Still's disease. | Week 4 to Week 54 |
| Change in the number/proportion of patients reporting problems by dimension of EQ-5D-3L(≥ 16 years). | To evaluate the health status in anakinra treated patients with Still's disease. | Week 4 to Week 54 |
| ACR30 response with absence of fever during the 7 days preceding the study visits over time. | To evaluate sustained efficacy of anakinra in patients responding to study drug. | Week 2 to Week 54 |
| To evaluate the occurrence of study drug discontinuation in anakinra treated patients with Still's disease. |
| Day 1 to Week 54 |
| Time of initiation of glucocorticoids tapering. | To evaluate glucocorticoids tapering in anakinra treated patients with Still's disease. | Week 2 to Week 54 |
| Percentage decrease of glucocorticoids dose for patients tapering their glucocorticoids dose. | To evaluate glucocorticoids tapering in anakinra treated patients with Still's disease. | Week 2 to Week 54 |
| Discontinuation of glucocorticoids. | To evaluate glucocorticoids tapering in anakinra treated patients with Still's disease. | Week 2 to Week 54 |
| - Occurrence of adverse events (AEs) (serious adverse events [SAEs] and non-SAEs). | To evaluate the safety of anakinra in patients with Still ́s disease. | Baseline to Week 58 |
| Occurrence of deaths | To evaluate the safety of anakinra in patients with Still ́s disease. | Baseline to Week 58 |
| Occurrence of AEs leading to study drug discontinuation at all study visits. | To evaluate the safety of anakinra in patients with Still ́s disease. | Baseline to Week 58 |
| Occurrence of vital signs changes from baseline, including blood pressure, heart rate, and body weight at all study visits up to Week 58 visit. Changes of height in patients younger than 19 years old. | To evaluate the safety of anakinra in patients with Still ́s disease. | Baseline to Week 58 |
| Occurrence of laboratory safety assessments changes over time. | To evaluate the safety of anakinra in patients with Still ́s disease. | Baseline to Week 58 |
| Occurrence of abnormal laboratory values. | To evaluate the safety of anakinra in patients with Still ́s disease. | Baseline to Week 58 |
| To evaluate immunogenicity of anakinra in patients with Still's disease. |
| Baseline, Weeks 2, 4, 12, 34, 54 and 58 |
| To evaluate the pharmacokinetic of anakinra in patients with Still's disease | Anakinra serum concentrations | Baseline, Weeks 1 and 2 |
| Kobe |
| Hyōgo |
| Japan |
| Kobe University Hospital | Kobe | Hyōgo | Japan |
| Yokohama City University Hospital (Hematology and Clinical Immunology) | Yokohama | Kanagawa | Japan |
| Shinshu University | Matsumoto | Nagano | Japan |
| Tokyo Medical And Dental University Hospital | Bunkyō-Ku | Tokyo | Japan |
| Tokyo Women's Medical University Hospital | Shinjuku-Ku | Tokyo | Japan |
| Gifu University Hospital | Gifu | Japan |
| D003240 |
| Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D001172 | Arthritis, Rheumatoid |
| D011506 | Proteins |
| D001685 | Biological Factors |