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临床试验/NCT05814159
NCT05814159进行中（未招募）3 期

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

Swedish Orphan Biovitrum8 个研究点 分布在 1 个国家目标入组 15 人开始时间: 2022年8月24日最近更新:
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适应症
干预措施
相关药物

试验速览

阶段
3 期
状态
进行中（未招募）
发起方
入组人数
15
试验地点
8
主要终点
An improvement of ≥ 30% from baseline in physician global assessment of disease activity (visual analogue scale [VAS]).
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

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]).

详细描述

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.

研究设计

研究类型
Interventional
分配方式
Randomized
干预模型
Parallel
主要目的
Treatment
盲法
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

盲法说明

Double blind

入排标准

年龄范围
8 Months 至 —（Child, Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •Male and female patients, 8 months of age or older with a body weight ≥ 10 kg
  • •Diagnosis of Still's disease
  • •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.
  • •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.
  • •The result of tuberculosis test within 8 weeks prior to enrollment is negative.

排除标准

  • •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.
  • •Narcotic analgesics within 24 hours prior to enrollment.
  • •Diaminodiphenyl sulfone within 1 week prior to enrollment or etanercept within 2 weeks prior to enrollment.
  • •Intraarticular, intramuscular, or intravenous administration of glucocorticoids within 72h(3 days) prior to enrollment, or intravenous immunoglobulin within 4 weeks prior to enrollment.
  • •Intravenous immunoglobulins with proven Still's disease modifying effect, leflunomide, infliximab, or adalimumab within 8 weeks prior to enrollment.
  • •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.
  • •Tocilizumab within 4 weeks prior to enrollment or any other immunomodulatory medications within 4 half-lives prior to enrollment.
  • •Rituximab within 13 weeks prior to enrollment.
  • •Canakinumab within 130 days 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
  • •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 .

研究组 & 干预措施

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.

干预措施: Anakinra (Drug)

Placebo

Placebo Comparator

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

干预措施: Placebo (Drug)

结局指标

主要结局

An improvement of ≥ 30% from baseline in physician global assessment of disease activity (visual analogue scale [VAS]).

时间窗: Week 2

ACR30 response with absence of fever attributable to the disease during the 7 days

An improvement of ≥ 30% from baseline in patient/parent global assessment of overall well-being (VAS).

时间窗: Week 2

ACR30 response with absence of fever attributable to the disease during the 7 days

An improvement of ≥ 30% from baseline in number of joints with active arthritis.

时间窗: Week 2

ACR30 response with absence of fever attributable to the disease during the 7 days

An improvement of ≥ 30% from baseline in number of joints with limitation of motion.

时间窗: Week 2

ACR30 response with absence of fever attributable to the disease during the 7 days

An improvement of ≥ 30% from baseline in assessment of physical function: Child health assessment questionnaire (CHAQ)/Stanford health assessment questionnaire (SHAQ).

时间窗: Week 2

ACR30 response with absence of fever attributable to the disease during the 7 days

An improvement of ≥ 30% from baseline in C-reactive protein (CRP) (mg/L).

时间窗: Week 2

ACR30 response with absence of fever attributable to the disease during the 7 days

次要结局

  • ACR70 response with absence of fever during the 24 hours preceding the evaluation visit at Week 1.(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).(Week 1)
  • Change in swelling joints count.(Week 4 to Week 54)
  • Change in CRP.(Week 4 to Week 54)
  • To evaluate the pharmacokinetic of anakinra in patients with Still's disease(Baseline, Weeks 1 and 2)
  • Change in ferritin.(Week 4 to Week 54)
  • Change in haemoglobin.(Week 4 to Week 54)
  • Change in platelets count.(Week 4 to Week 54)
  • Change in white blood cells count.(Week 4 to Week 54)
  • Absence of fever during the 24 hours preceding the evaluation visit at Week 1.(Week 1)
  • Absence of rash during the 24 hours preceding the evaluation visit at Week 1.(Week 1)
  • ACR30 response with absence of fever during the 24 hours preceding the evaluation visit at Week 1.(Week 1)
  • ACR50 response with absence of fever during the 24 hours preceding the evaluation visit at Week 1.(Week 1)
  • Change in physician global assessment of disease activity, measured on a VAS from no pain (0 mm) to very severe (100 mm).(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).(Week 1)
  • Change in tender joints count.(Week 4 to Week 54)
  • Change in the number/proportion of patients reporting problems by dimension of EQ-5D-Y Proxy (4-7 years).(Week 4 to Week 54)
  • Change in the number/proportion of patients reporting problems by dimension of EQ-5D-Y (8-15 years).(Week 4 to Week 54)
  • Change in the number/proportion of patients reporting problems by dimension of EQ-5D-3L (≥ 16 years).(Week 2)
  • Absence of fever during the 7 days preceding the visit.(Week 4 to Week 54)
  • Absence of rash during the 7 days preceding the visit.(Week 4 to Week 54)
  • ACR30 response with absence of fever during the 7 days preceding the visit.(Week 4 to Week 54)
  • ACR50 response with absence of fever during the 7 days preceding the visit.(Week 4 to Week 54)
  • ACR70 response with absence of fever during the 7 days preceding the visit.(Week 4 to Week 54)
  • ACR90 response with absence of fever during the 7 days preceding the visit.(Week 4 to Week 54)
  • Change in physician global assessment of disease activity (VAS).(Week 4 to Week 54)
  • Change in patient/parent global assessment of overall well-being (VAS).(Week 4 to Week 54)
  • Occurrence of AEs leading to study drug discontinuation at all study visits.(Baseline to Week 58)
  • Change in patient/parent global assessment of disease related pain (VAS).(Week 4 to Week 54)
  • Discontinuation of glucocorticoids.(Week 2 to Week 54)
  • Occurrence of inactive disease.(Week 8 to Week 54)
  • Occurrence of deaths(Baseline to Week 58)
  • Occurrence of laboratory safety assessments changes over time.(Baseline to Week 58)
  • Change in the number/proportion of patients reporting problems by dimension of EQ-5D-3L(≥ 16 years).(Week 4 to Week 54)
  • ACR30 response with absence of fever during the 7 days preceding the study visits over time.(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.(Week 2 to Week 54)
  • Percentage decrease of glucocorticoids dose for patients tapering their glucocorticoids dose.(Week 2 to Week 54)
  • - Occurrence of adverse events (AEs) (serious adverse events [SAEs] and non-SAEs).(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.(Baseline to Week 58)
  • Occurrence of abnormal laboratory values.(Baseline to Week 58)
  • To evaluate immunogenicity of anakinra in patients with Still's disease.(Baseline, Weeks 2, 4, 12, 34, 54 and 58)

研究者

发起方
Swedish Orphan Biovitrum
申办方类型
Industry
责任方
Sponsor

研究点 (8)

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