抗発作補助薬 (ASM) の実世界証拠 (RWE) 研究
現実世界において補助的なASMで治療されている局所発作発作の成人患者を対象とした12か月の前向き観察研究
The purpose of this study is to describe the effectiveness of the adjunctive ASM treatment on the clinical response, safety profile and quality of life of patients affected by focal onset seizures in a real-world setting.
調査の概要
詳細な説明
The aim of the study is to assess the effectiveness and safety of adjunctive therapy in a real-world setting of patients affected by focal-onset seizures who are eligible to start the treatment with ASM as adjunctive therapy according to the physician's judgment.
研究の種類
入学 (実際)
連絡先と場所
研究場所
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Milan、イタリア、20133
- Fondazione IRCCS Istituto Neurologico "Carlo Besta" U.O. Epilettologia Clinica e Sperimentale - Centro di Medicina del Sonno
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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
Adult patients with epilepsy having focal-onset seizures with or without secondary generalization not adequately controlled despite a history of treatment with at least 2 anti-seizure medications and who will meet all the inclusion and none of the exclusion study criteria.
The patients should be eligible to start a treatment with ASM as adjunctive therapy according to the physician's judgement, that must be clearly separated from the physician's decision to include the patient in the current study. The patients will be prospectively observed up to 12 months after having completed the related titration.
説明
Inclusion Criteria:
- Male and female patients of any ethnic origin ≥18 years old at baseline.
- Patients with diagnosis of focal-onset seizures with or without secondary generalization.
- Patients should have been eligible to start treatment with ASM as adjunctive therapy according to the physician's judgement prior to the inclusion.
- Patients should have clinical history of treatment failure with at least 2 ASMs.
- Patients using their seizure diary as part of their standard of care for at least 3 months prior to -the study entry (diary can be paper or electronic, filled in by patient and/or family members).
- Written informed consent (including data privacy consent) signed by the patient, legal guardian, or legally authorized representative prior to entering the study in accordance with the ICH GCP guidelines
Exclusion Criteria:
- Patients who meet any of the contraindications to the administration of adjunctive ASMs according to their approved SmPC.
- Progressive neurological disease, including degenerative CNS diseases and progressive tumors.
- Patients with unstable psychiatric diagnosis that may confound participants' ability to participate in the study or that may prevent completion of the protocol-specified assessments (e.g., in the judgement of the Investigator, pose an appreciable risk for suicide, including suicidal behavior and ideation within 6 months prior to enrollment, current psychotic disorder, acute mania).
- Evidence of clinically significant disease (e.g., cardiac, respiratory, gastrointestinal, renal disease) that in the opinion of the Investigator could affect the participant's safety or interfere with study assessments.
- Patients with substance abuse or dependence (except for caffeine and nicotine).
- Patients participating in any pharmacological or nonpharmacological interventional study within 30 days prior to baseline.
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：OTHER
- 時間の展望：PROSPECTIVE
コホートと介入
グループ/コホート |
介入・治療 |
|---|---|
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Adult patients with focal-onset seizures starting a treatment with ASM as adjunctive therapy
Adult patients having focal-onset seizures not adequately controlled despite a history of treatment with at least 2 ASM. The patients should be eligible to start a treatment with ASM as adjunctive therapy.The patients will be prospectively observed up to 12 months after having completed the related titration. |
ASM approved as adjunctive therapy |
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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Change in seizure frequency at 6 months of maintenance
時間枠：At 6 months of maintenance compared to baseline
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The effectiveness of adjunctive ASM is measured as change in seizure frequency at 6 months of maintenance compared to baseline |
At 6 months of maintenance compared to baseline
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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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Change in seizure frequency at 3, 9, 12 months of maintenance
時間枠：At 3, 9, 12 months of maintenance compared to baseline
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The effectiveness of adjunctive ASM is measured as change in seizure frequency at 3, 9, 12 months of maintenance compared to baseline |
At 3, 9, 12 months of maintenance compared to baseline
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50, 75, 90 Percent Responder rate
時間枠：At 3, 6, 9, and 12 months of maintenance phase.
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The effectiveness is measured as 50, 75, 90 Percent Responder rate |
At 3, 6, 9, and 12 months of maintenance phase.
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100 Percent Responder rate
時間枠：At 3, 6, 9, and 12 months of maintenance phase.
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The effectiveness is measured as number/percentage of seizure free patients |
At 3, 6, 9, and 12 months of maintenance phase.
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Retention rate
時間枠：At 3, 6, 9, and 12 months of maintenance phase.
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The effectiveness is measured as percentage of patients remaining in the study and on adjunctive therapy |
At 3, 6, 9, and 12 months of maintenance phase.
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Anxiety assessment
時間枠：At baseline, immediately after completion of titration, at 3 months, 6 months, and 12 months of maintenance phase.
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The assessment is measured by means of the Generalized Anxiety Disorder (GAD-7) scale. Scoring GAD-7 Anxiety Severity is calculated by assigning scores of 0, 1, 2, and 3 to the response categories, respectively, of "not at all," "several days," "more than half the days," and "nearly every day." GAD-7 total score for the seven items ranges from 0 to 21. 0-4: minimal anxiety 5-9: mild anxiety 10-14: moderate anxiety 15-21: severe anxiety. |
At baseline, immediately after completion of titration, at 3 months, 6 months, and 12 months of maintenance phase.
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Depression assessment
時間枠：At baseline, immediately after completion of titration, at 3 months, 6 months, and 12 months of maintenance phase.
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The assessment is measured through the Neurological Disorders Depression Inventory for Epilepsy (NDDI-E) scale. This is a validated screening tool for depression in patients with epilepsy that consists of a 6- item questionnaire. NDDI-E scores greater than 15 were considered positive for depression, as this score was previously shown to have a specificity of 90%, sensitivity of 81%, and positive predictive value of 0.62 for a diagnosis of major depression. |
At baseline, immediately after completion of titration, at 3 months, 6 months, and 12 months of maintenance phase.
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Quality of life (QOL)
時間枠：At baseline, immediately after completion of titration, at 3 months, 6 months, and 12 months of maintenance phase.
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The quality of life is measured by means the Quality Of Life In Epilepsy (QOLIE-31-P) questionnaire. This is a 38 questions survey of health-related quality of life for adults (18 years or older) with epilepsy. This version differs from the original QOLIE-31 (version 1) in the addition of questions about how much distress you feel about problems and worries related to epilepsy. This questionnaire should be completed only by the person who has epilepsy (not a relative or friend). Patients are asked to answer every question by circling the appropriate number (1, 2, 3...). |
At baseline, immediately after completion of titration, at 3 months, 6 months, and 12 months of maintenance phase.
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Cognitive assessment
時間枠：At baseline, immediately after completion of titration, at 3 months, 6 months, and 12 months of maintenance phase.
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The assessment of perceived cognitive deficits is measured through the Perceived Deficits Questionnaire (PDQ-5). The PDQ-5 assesses cognitive dysfunction in people with depression. This patient-reported questionnaire includes five items measuring attention/concentration, retrospective memory, prospective memory, and planning/organization over the past four weeks. The total score ranges from 0 to 20; higher scores indicate greater perceived cognitive dysfunction. |
At baseline, immediately after completion of titration, at 3 months, 6 months, and 12 months of maintenance phase.
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Adverse events (AEs)
時間枠：Through study completion, an average of 1 year
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Number of Adverse events (AEs) occurred (including AEs of special interest as Drug Reaction with Eosinophilia and Systemic Symptoms, rash/hypersensitivity, etc.). |
Through study completion, an average of 1 year
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協力者と研究者
研究記録日
主要日程の研究
研究開始 (実際)
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 (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
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