脊髄性筋萎縮症と診断されゾルゲンスマによる治療を受けたブラジル人患者における長期安全性研究 (ARISER)
オナセムノゲン・アベパルボベック（ゾルゲンスマ®）による治療を受けた脊髄性筋萎縮症（SMA）の診断が確定したブラジル人患者を対象とした長期安全性研究 - ARISER 研究
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
調査の概要
詳細な説明
This study is a non-interventional Post Authorization Safety Study (PASS) to evaluate long-term, real-world safety data of Brazilian pediatric patients diagnosed with SMA and treated with Onasemnogene Abeparvovec (Zolgensma®) for up to 15 years after the treatment. This study will support the benefit-risk assessment of Onasemnogene Abeparvovec in the approved indications and may also allow for detection of new safety signals and provide further guidance on the management of safety risks associated with Onasemnogene Abeparvovec to patients/caregivers, health care providers (HCPs) and treating physicians, as required by Brazilian Health Authority ANVISA as a conditional measure for granting Zolgensma®'s authorization.
研究の種類
入学 (推定)
連絡先と場所
研究連絡先
- 名前：Novartis Pharmaceuticals
- 電話番号：+41613241111
- メール：novartis.email@novartis.com
研究連絡先のバックアップ
- 名前：Novartis Pharmaceuticals
研究場所
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Paraná
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Curitiba、Paraná、ブラジル、81520-060
- 募集
- Novartis Investigative Site
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São Paulo
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São Paulo、São Paulo、ブラジル、05403-000
- 募集
- Novartis Investigative Site
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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
サンプリング方法
調査対象母集団
The study population will consist of approximately 50 Brazilian pediatric patients with SMA who were treated with Onasemnogene Abeparvovec (Zolgensma®) in the commercial setting, the closed MAP, or the phase IV OFELIA trial. Patients will be recruited in up to 3 centers in Brazil, over one year of recruitment.
説明
Inclusion Criteria:
- Subject's parent or legal guardian has provided signed eICF.
- Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1 gene, and a clinical diagnosis of SMA Type 1 or up to 3 copies of the SMN2 gene.
- Subject treated* with Onasemnogene Abeparvovec (Zolgensma®) prior to enrolling in this study.
Subjects treated with nusinersen or risdiplam prior to Onasemnogene Abeparvovec (Zolgensma®) can be enrolled if currently not receiving it.
*Subjects can be enrolled in this study on the day treated with Onasemnogene Abeparvovec (Zolgensma®) or if prior medical history is available to complete all assessments retrospectively, in accordance with local ethical requirements.
- Subject and parent/guardian are willing and able to comply with the phone contacts through the course of the study
Exclusion Criteria:
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Patients currently enrolled in any interventional clinical trial** other than the phase IV OFELIA trial will be excluded from the study.
- Subjects who were enrolled in a clinical trial (independently of the disease indication and interventional treatment) but are not currently enrolled, can be included in this study.
During the follow-up, subjects who enroll any clinical trial with pharmacological intervention will discontinue from this study.
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
- 観測モデル：COHORT
- 時間の展望：OTHER
コホートと介入
グループ/コホート |
介入・治療 |
|---|---|
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Spinal Muscular Atrophy Patients
Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec |
Retrospective and prospective observational study. There is no treatment allocation.
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 |
|---|---|---|
|
Incidence and severity of treatment-emergent SAEs
時間枠：Up to 5 years
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Incidence and severity of treatment-emergent Serious AEs (SAEs) |
Up to 5 years
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Number of patients who experience at least one AESI and number of patients by AESI
時間枠：Up to 15 years
|
Adverse Event of Special Interest: thrombocytopenia, hepatotoxicity, thrombotic microangiopathy (TMA), dorsal root ganglia toxicity, cardiac AEs, new malignancies, new incidence of neurologic, autoimmune, and hematologic disorder |
Up to 15 years
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Number of patients who have survived and have not required permanent ventilatory assistance
時間枠：Up to 15 years
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Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation. |
Up to 15 years
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Time until death or the need for permanent ventilatory assistance
時間枠：Up to 15 years
|
Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation. |
Up to 15 years
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Number of patients who achieve each Developmental Motor Milestone
時間枠：Up to 15 years
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Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone. |
Up to 15 years
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Number of patients who achieve each WHO Developmental Milestone within age percentiles
時間枠：Up to 15 years
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Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone. |
Up to 15 years
|
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：Novartis Pharmaceuticals、Novartis Pharmaceuticals
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
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?
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