Single Patient Use of Tocilizumab in Systemic Onset Juvenile Idiopathic Arthritis
Single Patient Use of Tocilizumab for Treatment of Steroid Dependent, Active Systemic Onset Juvenile Idiopathic Arthritis
調査の概要
詳細な説明
Systemic onset Juvenile Idiopathic Arthritis (soJIA) is a type of arthritis (inflammation of the joints) that occurs with other symptoms including fever, swollen lymph nodes (glands), rash, and body aches. Because soJIA can be difficult to treat, children with soJIA can have severe problems from long-term use of steroids (prednisone). These problems include low bone density (weak bones), fractures, failure to grow properly, and large weight gain. The arthritis that occurs in soJIA often causes damage to many joints. This can make it hard to move around or do basic tasks like dressing. Also, a life-threatening illness called Macrophage Activation Syndrome (MAS) can occur when starting, stopping, or changing drugs that are used to treat soJIA.
SoJIA can be hard to treat and many children with soJIA do not respond to drugs that work for other kinds of arthritis. Research doctors have studied a chemical signal called IL-6 that the body uses to manage inflammation. This signal has been found to be very high in patients with active soJIA. A drug called tocilizumab (TCZ) has been designed to block IL-6. For about 6 years, TCZ has been tested in Japan for treating soJIA. It is now being tested in studies in the United States. These studies can have very strict rules for enrolling patients. This trial is a single-patient research study for a subject who otherwise does not meet the rules for enrollment in ongoing trials.
研究の種類
入学 (実際)
段階
- 適用できない
連絡先と場所
研究場所
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Massachusetts
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Boston、Massachusetts、アメリカ、02111
- Tufts Medical Center/Floating Hospital for Children
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参加基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
Inclusion Criteria:
- Systemic Juvenile Idiopathic Arthritis according to ILAR criteria (2001)
- Duration of disease ≥ 6 months since onset
- Presence of active disease as determined by the presence of at least 5 active joints, OR at least 2 active joints if receiving prednisone at a dose > 0.2 mg/kg/day or > 10 mg/day (whichever is less)
- Incomplete prior response to methotrexate treatment for at least 3 months at a minimum dose of 15 mg/M2/week, or intolerance to methotrexate
- Discontinued treatment with other biologics prior to first tocilizumab infusion, for approximately two pharmacokinetic half-lives as per specific biologic (e.g. 48 hours for anakinra, 7 days for etanercept)
- Not receiving corticosteroids, OR taking oral corticosteroids and the dose has remained stable for 1 week prior to the first tocilizumab infusion at ≤ 2 mg/kg/day prednisone or prednisolone and no more than 80 mg/day
Exclusion Criteria:
- Concomitant administration of biologic therapies
- Serum creatinine >1.5 ULN (upper limits normal)
- AST or ALT > 1.5 ULN
- Total bilirubin > 1.3 mg/dL
- Platelet count < LLN (lower limits normal)
- Hemoglobin < 6.0 g/dL
- WBC count < 5,000/mm3
- Neutrophil count < 2,000/ mm3
- Fibrinogen < LLN
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：処理
- 割り当て：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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実験的：Tocilizumab
Single arm study - treatment only
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Initial therapy: Tocilizumab dosed by body weight (8mg/kg based on body weight ≥ 30kg) given by intravenous infusion every two weeks for 12 weeks. Extension of therapy: Continuation of treatment with tocilizumab at 8mg/kg by body weight given by intravenous infusion every 2 weeks based upon achievement of Primary Objective by week 12, OR continuation of treatment with escalation of tocilizumab dose to 12mg/kg by body weight, given by intravenous infusion every two weeks, for failure to achieve ACR JIA30 at 12 weeks or ACR JIA50 response at any time after week 16.
他の名前：
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この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Efficacy of Tocilizumab as Defined by Presence of an Equal to or Greater Than 30% Improvement in JIA Core Set (i.e. ACR JIA30 Response)
時間枠：At week 12 of treatment versus week 0 (pretreatment)
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At week 12 of treatment versus week 0 (pretreatment)
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Efficacy of Tocilizumab as Defined by Reduction of Oral Prednisone Dose by at Least 20%, or to Less Than 0.5mg/kg/Day, Whichever is of Lesser Daily Dose, While Maintaining an ACR JIA30 Response
時間枠：At weeks 12 and 16 of treatment versus week 0 (pretreatment)
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At weeks 12 and 16 of treatment versus week 0 (pretreatment)
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Number of Participants With at Least One Adverse Event
時間枠：Ongoing, throughout 24 month study period
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To evaluate the safety of tocilizumab administration in this subject
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Ongoing, throughout 24 month study period
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Measurement of Laboratory Parameters of Active Disease, Specifically C-reactive Protein, Hemoglobin, Platelets, White Blood Cell Count, Ferritin, Immunoglobulins.
時間枠：At weeks 8, 12, and 16 of treatment, and every 8-12 weeks thereafter
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To assess normalization of laboratory parameters of active disease, specifically C-reactive protein, hemoglobin, platelets, white blood cell
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At weeks 8, 12, and 16 of treatment, and every 8-12 weeks thereafter
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Measurement of Sustained Clinical Response to Tocilizumab, Including Active Joint Count, Joints With Limited Range of Motion, and Absence of Fever or Rash.
時間枠：At weeks 8, 12, 16 of treatment, and every 8 weeks thereafter
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To assess sustained clinical response to tocilizumab, including active joint count, joints with limited range of motion, and absence of fever
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At weeks 8, 12, 16 of treatment, and every 8 weeks thereafter
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協力者と研究者
出版物と役立つリンク
研究記録日
主要日程の研究
研究開始
一次修了 (実際)
研究の完了 (実際)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (見積もり)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
キーワード
その他の研究ID番号
- TMC-PRHEU-TCZ-01
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。