A Study of Anakinra in Japanese Patients With Still's Disease (SJIA and AOSD)

NCT ID: NCT05814159

Last Updated: 2025-03-05

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-08-24
• Study Completion Date: 2026-06-24

Brief Summary

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]).

Detailed Description

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.

Conditions

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Anakinra

100 mg/day or 2 mg/kg/day of subcutaneous anakinra for those with a body weight ≥50 kg or \<50 kg, respectively.

Group Type: EXPERIMENTAL

Anakinra

Intervention Type: DRUG

sub cutaneous daily injection

Placebo

Corresponding volume to 100 mg/day or 2 mg/kg/day

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

sub cutaneous daily injection

Interventions

Anakinra

sub cutaneous daily injection

Intervention Type: DRUG

Placebo

sub cutaneous daily injection

Intervention Type: DRUG

Other Intervention Names

Kineret

Eligibility Criteria

Inclusion Criteria

1. Male and female patients, 8 months of age or older with a body weight ≥ 10 kg

2. Diagnosis of Still's disease

3. If < 16 years of age at disease onset, the diagnosis is madeaccording to adapted ILAR criteria i.e., CARRA criteria for SJIA. If ≥ 16 years of age at disease onset, the diagnosis is made according to Yamaguchi criteria for AOSD.

4. Active disease confirmed by the following three signs and symptoms. a. Active arthritis in ≥ 1 joint. b. CRP > 30 mg/L. c. At least one fever episode (≥ 38.0 degree Celsius) attributable to the disease within one week before enrollment.

5. The result of tuberculosis test within 8 weeks prior to enrollment is negative.

Exclusion Criteria

1. 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.

2. Use of the following therapies prior to enrollment.

1. Narcotic analgesics within 24 hours prior to enrollment.

2. Diaminodiphenyl sulfone within 1 week prior to enrollment or etanercept within 2 weeks prior to enrollment.

3. Intraarticular, intramuscular, or intravenous administration of glucocorticoids within 72h(3 days) prior to enrollment, or intravenous immunoglobulin within 4 weeks prior to enrollment.

4. Intravenous immunoglobulins with proven Still's disease modifying effect, leflunomide, infliximab, or adalimumab within 8 weeks prior to enrollment.

5. Thalidomide within 72h(3 days) prior to enrollment, cyclosporine within 5 weeks prior to enrollment, mycophenolate mofetil within 1 week prior to enrollment, 6-mercaptopurine within 48h(2 days) prior to enrollment, azathioprine within 72h(3 days) prior to enrollment, cyclophosphamide within 96h(4 days) prior to enrollment, chlorambucil (not approved inJapan) within 48h(2 days) prior to enrollment, or any other immunosuppressants within 12 weeks prior to enrollment.

6. Tocilizumab within 4 weeks prior to enrollment or any other immunomodulatory medications within 4 half-lives prior to enrollment.

7. Rituximab within 13 weeks prior to enrollment.

8. Canakinumab within 130 days prior to enrollment

3. Live vaccines within 4 weeks prior to enrollment.

4. 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.

5. Clinical evidence of liver disease or liver injury as indicated by presence of abnormal liver tests.

6. Presence of severe chronic kidney disease (CKD) grades 4 and 5.

7. Presence of neutropenia (absolute neutrophil count [ANC] < 1.5 x 10^9/L).

8. Presence of thrombocytopenia (platelets count < 100 x 10^9/L).

9. Presence or suspicion of MAS at baseline.

10. 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 .

• Minimum Eligible Age: 8 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

CMIC Co, Ltd. Japan

INDUSTRY

Sponsor Role: collaborator

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Masaaki Mori, MD

Role: PRINCIPAL_INVESTIGATOR

St. Marianna University Hospital

Locations

Fukushima Medical University Hospital

Fukushima, Fukushima, Japan

Hyogo Prefectural Kobe Children's Hospital

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

Countries

Japan

Other Identifiers

Sobi.ANAKIN-303

Identifier Type: -

Identifier Source: org_study_id