オナセムノ遺伝子Abeparvovec-xioiを投与された患者の長期追跡調査
AVXS-101を投与された脊髄性筋萎縮症の臨床試験における患者の長期追跡調査
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
調査の概要
研究の種類
入学 (実際)
段階
- フェーズ 3
連絡先と場所
研究場所
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California
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Palo Alto、California、アメリカ、94304
- Stanford University Medical Center
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Colorado
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Aurora、Colorado、アメリカ、80045
- Children's Hospital Colorado
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Illinois
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Chicago、Illinois、アメリカ、60611
- Ann Robert H. Lurie Children's Hospital of Chicago
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Maryland
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Baltimore、Maryland、アメリカ、21287
- John Hopkins Hospital - David M. Rubenstein Child Health Building
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Massachusetts
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Boston、Massachusetts、アメリカ、02114
- Massachusetts General Hospital
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Boston、Massachusetts、アメリカ、02115
- Boston Children's Hospital
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Michigan
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Grand Rapids、Michigan、アメリカ、49503
- Spectrum Health Hospitals Helen DeVos Children's Hospital
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Missouri
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St Louis、Missouri、アメリカ、63110
- Washington Unviersity School of Medicine in Saint Louis
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New York
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New York、New York、アメリカ、10032
- Columbia University Medical Center
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North Carolina
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Durham、North Carolina、アメリカ、27713
- Duke University
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Ohio
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Columbus、Ohio、アメリカ、43205
- Nationwide Children's Hospital
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Pennsylvania
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Philadelphia、Pennsylvania、アメリカ、19104
- Children's Hospital of Philadelphia
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Strasburg、Pennsylvania、アメリカ、17579
- Clinic for Special Children
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Texas
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Dallas、Texas、アメリカ、75235
- Children's Health Specialty Center Dallas Campus
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Utah
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Salt Lake City、Utah、アメリカ、84112
- University of Utah Health
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Virginia
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Norfolk、Virginia、アメリカ、23507
- Children's Hospital of The King's Daughters
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Richmond、Virginia、アメリカ、23298
- Virginia Commonwealth University
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Wisconsin
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Madison、Wisconsin、アメリカ、53792
- University of Wisconsin, Madison
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Newcastle upon Tyne、イギリス、NE1 4LP
- The Newcastle Upon Tyne Hospitals NHS Foundation Trust
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London
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London、London、イギリス、WC1N 3JH
- Great Ormond Street Hospital for Children NHS Foundation Trust
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Genova、イタリア、16147
- Instituto Gianninia Gaslini
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Milan、イタリア、20133
- Istituto Neurologico di Ricerca
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Milan、イタリア、20122
- Universita Degli Studi Di Milano
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Roma
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Roma、Roma、イタリア、00168
- Fondazione Policlinico Universitario Agostino Gemelli
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New South Wales
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Randwick、New South Wales、オーストラリア、2145
- Sydney Children's Hospital
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Ontario
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Ottawa、Ontario、カナダ、K1H8L1
- Children's Hospital of Eastern Ontario Research Institute
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Paris、フランス、75012
- Hôpital Armand Trousseau
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Ghent、ベルギー、9000
- Universitair Ziekenhuis Gent
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Liège
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Liège、Liège、ベルギー、B-4000
- Centre de Référence des Maladies Neuromusculaires
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Taipei、台湾、10048
- National Taiwan University Hospital
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Tokyo、日本、162-8666
- Tokyo Women's Medical University Hospital
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参加基準
適格基準
就学可能な年齢
- 子
- 大人
- 高齢者
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:
- Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study
- Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
Exclusion Criteria:
- Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety 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
- 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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他の：Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi
Participants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene. |
Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.
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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身長測定値のベースラインからの変化
時間枠：5年まで
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5年まで
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体重測定値のベースラインからの変化
時間枠：5年まで
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5年まで
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併用薬全体および薬の種類別の参加者数
時間枠：5年まで
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5年まで
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他の SMA 療法の参加者全体および薬剤の種類別の数
時間枠：6年生から15年生
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6年生から15年生
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Number of Participants Who Reach Developmental Milestones
時間枠：Up to 5 years
|
Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone. |
Up to 5 years
|
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Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
時間枠：Up to 5 years
|
The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments rated from 0 (unable to perform functional task) to 2 (able to perform functional task unassisted). Higher scores on the total scale of 0-66 indicates higher levels of motor ability. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support
時間枠：Up to 15 years
|
Participants will receive pulmonary assessments by a pulmonologist or appropriate clinician. Respiratory device data will be reviewed for participants receiving non-invasive ventilatory support. |
Up to 15 years
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Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support
時間枠：Up to 5 years
|
Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question. |
Up to 5 years
|
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Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings
時間枠：Up to 5 years
|
The physical examination includes review of the following systems: head, ears, eyes, nose and throat, lungs/thorax, cardiovascular, abdomen, musculoskeletal, neurologic, dermatologic, lymphatic, and genitourinary. In addition, visual inspection of the spine, back, shoulders, and hips looking for spinal curvature and asymmetry will be carried out. Joints will be assessed for loss of mobility and contractures. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements
時間枠：Up to 5 years
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Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments
時間枠：Up to 5 years
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Blood samples will be collected for hematology (including complete blood cell count) and chemistry. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments
時間枠：Up to 5 years
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Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results
時間枠：Year 6 to Year 15
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The observational phase questionnaire includes 7 yes/no questions. Observation categories include: adverse events, hospitalizations, concomitant medications, ventilatory support and feeding support. |
Year 6 to Year 15
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Number of Participants Who Experience at Least One Serious Adverse Event (SAE)
時間枠：Up to 15 years
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An SAE is defined as any adverse event (appearance of [or worsening of any pre existing]) undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria:
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Up to 15 years
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Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)
時間枠：Up to 15 years
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An AESI is defined as an AE occurring during any study phase that fulfills one of the following criteria:
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Up to 15 years
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Change From Baseline in Bayley Scales of Infant and Toddler Development
時間枠：Up to 42 months, 15 days of age
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Third Edition (Bayley-III) to be performed in all patients up to 42 months, 15 days of age. |
Up to 42 months, 15 days of age
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Change From Baseline in Revised Upper Limb Module (RULM) Score
時間枠：Up to 5 years
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RULM score is based on a scale from 0 to 37 where lower scores reflect poorer upper limb functional ability. |
Up to 5 years
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Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older
時間枠：Up to 5 years
|
The Cogstate Computerized Cognitive Battery consists of the Identification Test (scored 0 (best) to 1.5708 (worst)), the International Shopping List Test (scored 0 (worst) to 999 (best)), the International Shopping List Test-Delayed Recall (scored 0 (worst) to 999 (best)), the One Card Learning Test (scored 0 (worst) to 1.5708 (best)), and the One Back Test (scored 0 (worst) to 1.5708 (best)). |
Up to 5 years
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Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age
時間枠：Up to 5 years
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The CELF-5 Following Directions and Sentence Repetition subtests use scoring that varies based on age, but will be administered to participants 5-21 years of age. The Following Directions subtest will be scored from 0-33 with higher score being more advanced and the Recalling Sentences subtest will be scored from 0-78 with higher score being more advanced. |
Up to 5 years
|
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Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)
時間枠：Up to 5 years
|
ACEND score is based on a scale from 1 to 41 where higher scores represent a better caregiver experience |
Up to 5 years
|
協力者と研究者
スポンサー
捜査官
- スタディチェア：Sitra Tauscher-Wisniewski, MD、Novartis Gene Therapies, Inc.
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
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
- AVXS-101-LT-002
- 2019-002611-26 (EudraCT番号)
- 205305 (その他の識別子：JapicCTI)
- COAV101A12103 (その他の識別子：Novartis Pharmaceuticals)
- 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
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
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
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。