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A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients

2026年9月14日 更新者：Novartis Pharmaceuticals

Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)

This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.

This is a non-interventional study and does not impose a therapy protocol, diagnostic/therapeutic procedure, or a visit schedule. Patients will be treated according to the Japan package insert. Data will be collected using a case registration form (CRF) completed by the investigator at each study site.

調査の概要

状態

まだ募集していません

条件

研究の種類

観察的

入学 (推定)

80

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究連絡先

研究連絡先のバックアップ

  • 名前：Novartis Pharmaceuticals

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

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

いいえ

サンプリング方法

非確率サンプル

調査対象母集団

SMA patients administered onasemnogene abeparvovec by intrathecal injection in Japan.

説明

Inclusion criteria:

• All patients treated with onasemnogene abeparvovec.

Exclusion criteria:

• Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

コホートと介入

グループ/コホート
Onasemnogene Abeparvovec Intrathecal Group
SMA patients treated with onasemnogene abeparvovec intrathecal injection in Japan.

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
Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs)
時間枠：Up to approximately 4 years and 5 months
Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs
時間枠：Up to approximately 4 years and 5 months
Incidence based on the person-year method will be calculated.
Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
時間枠：Up to approximately 4 years and 5 months
Incidence based on the person-year method will be calculated.
Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs
時間枠：Up to approximately 4 years and 5 months
Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs Corresponding to the Safety Specifications
時間枠：Up to approximately 4 years and 5 months
Up to approximately 4 years and 5 months
Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications
時間枠：Up to approximately 4 years and 5 months
Safety specifications include hepatotoxicity, transient thrombocytopenia, thrombotic microangiopathy, cardiac adverse events, dorsal root ganglia toxicity/peripheral sensory neuropathy, tumorigenicity due to chromosomal integration, and long-term monitoring of gene therapy.
Up to approximately 4 years and 5 months

二次結果の測定

結果測定
メジャーの説明
時間枠
Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score
時間枠：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Change From Baseline in Revised Upper Limb Module (RULM) Score
時間枠：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

RULM is a tool used to assess upper limb function in SMA patients. It consists of 20 items in total. The first entry item classifies overall functional ability and is not scored. The remaining 19 tasks are scored with 1 task scored as can/cannot (1 is the highest score) and 18 tasks rated on a 3-point scale as follows:

  • 0 - unable to perform the task
  • 1 - able to perform the task with some modification
  • 2 - able to perform the task with no difficulty The total RULM score ranges from 0 to 37 with higher scores indicating better upper limb function.
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score
時間枠：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
CHOP-INTEND is a tool used to measure the motor abilities in infants with SMA. It consists of 16 items scored on a scale from 0 (no response/unable to perform the movement) to 4 (complete response/able to perform the task). CHOP-Intend total score ranges from 0 to 64 with higher scores indicating higher motor function.
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Change From Baseline in Clinical Global Impression-Severity (CGI-S) Score
時間枠：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

The CGI-S scale is a physician-rated measure used to assess the severity of a patient's illness. Severity of illness is assessed with consideration for the overall clinical experience of patients of the same age. The CGI-S scores range from:

  • 0 = Not evaluated
  • 1 = Normal, not at all ill
  • 2 = Borderline normal and ill
  • 3 = Mildly ill
  • 4 = Moderately ill
  • 5 = Markedly ill
  • 6 = Severely ill
  • 7 = Among the most extremely ill
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Number of Patients Categorized by Clinical Global Impression-Improvement (CGI-I) Score
時間枠：6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

The CGI-I scale is a physician-rated measure used to assess a patient's overall clinical status. The patient's overall clinical status is scored on a 7-point scale of the extent of change relative to before treatment initiation as follows:

  • 1 = Very much improved
  • 2 = Much improved
  • 3 = Minimally improved
  • 4 = No change
  • 5 = Minimally worse
  • 6 = Much worse
  • 7 = Very much worse Number of patients and their 95% confidence intervals (CIs) will be calculated for two categories: improved (CGI-I score: 1 to 3) and unchanged or worsened (CGI-I score: 4 to 7).
6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Number of Patients Achieving Each Developmental Milestone
時間枠：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

Developmental milestones include:

  • Sitting without support
  • Hands-and-knees crawling
  • Standing with assistance
  • Walking with assistance
  • Standing alone
  • Walking alone
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Number of Patients Maintaining Each Developmental Milestone
時間枠：Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

Novartis Pharmaceuticals

捜査官

  • スタディディレクター：Novartis Pharmaceuticals、Novartis Pharmaceuticals

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (推定)

2026年10月30日

一次修了 (推定)

2031年3月31日

研究の完了 (推定)

2031年3月31日

試験登録日

最初に提出

2026年8月11日

QC基準を満たした最初の提出物

2026年8月11日

最初の投稿 (実際)

2026年8月17日

学習記録の更新

投稿された最後の更新 (実際)

2026年9月17日

QC基準を満たした最後の更新が送信されました

2026年9月14日

最終確認日

2026年9月1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

  • COAV101B1401

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?

いいえ

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