A Study to Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, and Pharmacodynamics of Satralizumab in Pediatric Patients With Aquaporin-4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD) (SAkuraSun)
A Phase III, Multicenter, Open-Label, Uncontrolled Study to Evaluate Pharmacokinetics, Efficacy, Safety, Tolerability, and Pharmacodynamics of Satralizumab in Pediatric Patients With AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disorder (NMOSD)
This study will primarily evaluate the pharmacokinetics of satralizumab in pediatric patients aged 2-11 years with anti-aquaporin-4 (AQP4) antibody seropositive neuromyelitis optica spectrum disorder (NMOSD). Efficacy, safety, tolerability, and pharmacodynamics will be evaluated in a descriptive manner, given the small number of patients who will be enrolled in this study.
調査の概要
研究の種類
入学 (推定)
段階
- フェーズ 3
連絡先と場所
研究連絡先
- 名前：Reference Study ID Number: WN41733 https://forpatients.roche.com/ No attachments to email below.
- 電話番号：888-662-6728 (U.S.)
- メール：global-roche-genentech-trials@gene.com
研究連絡先のバックアップ
- 名前：Fastest response: use the inquiry form. https://www.gene.com/contact-us/submit-medical-inquiry
- メール：global.medical_information@roche.com
研究場所
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Colorado
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Denver、Colorado、アメリカ、80218-1007
- 募集
- Children's Hospital Colorado.
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Ciudad Autonoma Buenos Aires、アルゼンチン、C1249ABN
- 募集
- Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
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Córdoba、アルゼンチン、X5004FHP
- 募集
- Clinica Universitaria Reina Fabiola
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London、イギリス、WC1N 3JH
- 募集
- Great Ormond Street Hospital for Children
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Lazio
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Rome、Lazio、イタリア、00165
- 募集
- IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
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Lombardy
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Pavia、Lombardy、イタリア、27100
- 募集
- Fondazione Istituto Neurologico Mondino IRCCS
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Kocaeli、トルコ（Türkiye）、6810
- 募集
- Kocaeli University Research and Application Hospit
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Val-de-Marne
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Le Kremlin-Bicêtre、Val-de-Marne、フランス、94275
- 募集
- Centre Hospitalier Universitaire de Bicêtre
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Gdansk、ポーランド、80-214
- 積極的、募集していない
- Uniwersyteckie Centrum Kliniczne
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Warsaw、ポーランド、04-730
- 募集
- Instytut "Pomnik - Centrum Zdrowia Dziecka"
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Mexico CITY (federal District)
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DF、Mexico CITY (federal District)、メキシコ
- 募集
- Grupo Medico Camino
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Mexico City、Mexico CITY (federal District)、メキシコ、06720
- 募集
- Hospital Infantil de Mexico Federico Gomez
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Guangdong
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Guangzhou、Guangdong、中国、510120
- 募集
- Guangzhou Women and Children's Medical Center
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Shanghai Municipality
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Shanghai、Shanghai Municipality、中国、201102
- 募集
- Children's Hospital of Fudan University
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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:
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Age at screening 2-11 years, inclusive
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Body weight at screening >=10 kg
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For female patients of childbearing potential (postmenarchal): agreement to either remain completely abstinent (refrain from heterosexual intercourse) or to use a reliable means of contraception
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Diagnosed as having NMOSD with AQP4 antibody seropositive status as defined by the Wingerchuk 2015 criteria Clinical evidence of at least one documented attack (including first attack) in the last year prior to screening:
- Documented history of a positive anti-AQP4 antibody test or a positive anti-AQP4 antibody test at screening;
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Neurological stability for >=30 days prior to baseline
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Expanded Disability Status Scale (EDSS) 0 to 6.5
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For participants receiving a baseline immunosuppressant treatment and planning to continue on these therapies, treatment must be at stable dose for 4 weeks prior to baseline
Exclusion Criteria:
- Pregnancy or lactation
- Evidence of other demyelinating disease mimicking NMOSD
- Active or presence of recurrent bacterial, viral, fungal, mycobacterial infection, or other infection at baseline
- Evidence of chronic active hepatitis B or C
- Evidence of untreated latent or active tuberculosis (TB)
- Receipt of a live or live-attenuated vaccine within 6 weeks prior to baseline
- History of severe allergic reaction to a biologic agent
- IVIg or subcutaneous Ig (SCIg) within 4 weeks prior to enrollment/Day 1
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：非ランダム化
- 介入モデル：並列代入
- 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：Cohort 1: Participants with body weight ≥10kg to <20kg
Satralizumab will be administered SC Q6W in a cohort of at least 2 evaluable patients |
Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period. |
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実験的：Cohort 2 Participants with body weight ≥20kg to <40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter. |
Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period. |
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実験的：Cohort 3 Participants with body weight ≥40kg
Satralizumab will be administered SC at Weeks 0, 2, 4, and Q4W thereafter. |
Participants will receive satralizumab treatment for a minimum of 48 weeks and then will have the opportunity to enter an optional satralizumab extension (OSE) period. |
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 |
時間枠 |
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Summary of Observed Serum Concentration of Satralizumab
時間枠：Week 48
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Week 48
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Apparent Clearance [CL/F] of Satralizumab
時間枠：Week 48
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Week 48
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Apparent Volume of Distribution [V/F] of Satralizumab
時間枠：Week 48
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Week 48
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Area Under the Concentration-time Curve [AUC] of Satralizumab
時間枠：Week 48
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Week 48
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二次結果の測定
結果測定 |
時間枠 |
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24週目および48週目の拡張障害ステータススケール（EDSS）のベースラインからの変化
時間枠：ベースライン、24週目、48週目
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ベースライン、24週目、48週目
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Proportion of Relapse-free Participants by Week 48
時間枠：Week 48
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Week 48
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Annualized Relapse Rate (ARR), defined as the average number of relapses for each year of the study
時間枠：Week 48
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Week 48
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Time to First Relapse (TFR) after enrollment, defined as the time from enrollment until the first occurrence of relapse, as determined by the investigator
時間枠：Week 48
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Week 48
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Time to Relapse Requiring Rescue Therapy
時間枠：Week 48
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Week 48
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Change from Baseline in Visual Acuity at Weeks 24 and 48
時間枠：Baseline, Week 24, Week 48
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Baseline, Week 24, Week 48
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Change from Baseline in FACES Pain Rating Scale at Weeks 24 and 48
時間枠：Baseline, Week 24, Week 48
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Baseline, Week 24, Week 48
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Change from Baseline in EuroQol 5-Dimension, Youth (EQ-5D-Y) Score and its Proxy at Weeks 24 and 48
時間枠：Baseline, Week 24, Week 48
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Baseline, Week 24, Week 48
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Incidence and Severity of Adverse Events
時間枠：Week 48
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Week 48
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協力者と研究者
スポンサー
捜査官
- スタディディレクター：Clinical Trials、Hoffmann-La Roche
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 (実際)
一次修了 (推定)
研究の完了 (推定)
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 (実際)
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
追加の関連 MeSH 用語
その他の研究ID番号
- WN41733
- 2019-004092-39 (EudraCT番号)
- 2023-507817-85-00 (Ctis)
- 2023 (米国 NIH グラント/契約)
- 2019 (米国 NIH グラント/契約)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
IPD プランの説明
For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
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