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临床试验/NCT05644899
NCT05644899已完成不适用

Interlaminar Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy: a Retrospective Analysis of More Than 500 Consecutive Administrations

Fondazione Policlinico Universitario Agostino Gemelli IRCCS1 个研究点 分布在 1 个国家目标入组 51 人开始时间: 2022年12月16日最近更新:
适应症

试验速览

阶段
不适用
状态
已完成
入组人数
51
试验地点
1
主要终点
Number of successful administrations

研究概览

简要总结

Nusinersen (Spinraza, Biogen Inc, Boston, MA), the first treatment approved by FDA and EMA for all Spinal Muscular Atrophy (SMA) subtypes, is an antisense oligonucleotide that is administered intrathecally through a lumbar puncture. This procedure can be challenging in some adults with intermediate and late onset SMA (types II-IV) frequently presenting scoliosis secondary to neuromuscular weakness and often treated with spinal instrumentation to prevent worsening deformities.

In such patients, in order to access the intrathecal space, US guidance and/or assistance have been recently proposed as useful and successful tool. The US guidance and/or assistance have been associated to a high success rate, a reduction of number of attempts and needle passes to obtain a successful anesthesia. A reduced risk of adverse events (AEs), such as post dural puncture headache (PDPH) and low back pain (LBP), and low patient satisfaction often associated with multiple needle punctures was also reported.

Aim of this retrospective study was to report the efficacy, evaluated as rate of the successful procedures and subsequent delivery of nusinersen within the subarachnoid space, the number of attempts, the procedure time and the adverse events (AEs) of interlaminar intrathecal nusinersen administration using either ultrasound assistance or the landmark-based technique in a historical cohort of 51 adult SMA patients.

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Retrospective

入排标准

年龄范围
18 Years 至 —(Adult, Older Adult)
性别
All
接受健康志愿者

入选标准

  • A genetically confirmed diagnosis of SMA
  • Treatment with nusinersen

排除标准

  • congenital coagulopathy,
  • localized infections,
  • increased intracranial pressure.

结局指标

主要结局

Number of successful administrations

时间窗: During the procedure

Defined as a confirmation of the CSF flow through the spinal needle and subsequent administration of whole drug

Number of attempts

时间窗: During the procedure

Defined as the number of needle insertions through the skin surface

Procedure time

时间窗: During the procedure

The time in minutes from the start of US imaging or the palpation of superior aspect of iliac crest to visualization of CSF flow

Number of technical success procedures

时间窗: During the procedure

Defined as a successful intrathecal administration with equal or less than 4 attempts

Adverse events (AEs)

时间窗: In the first 72 hours after the procedure

Defined as mild AEs (Post Dural Puncture Headache, Low Back Pain) or severe AEs (spinal cord injury/epidural hematoma, abdominal organ lesions)

次要结局

未报告次要终点

研究者

申办方类型
Other
责任方
Sponsor

研究点 (1)

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