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Clinical Trials/NCT05644899
NCT05644899
Completed
Not Applicable

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 site in 1 country51 target enrollmentDecember 16, 2022

Overview

Phase
Not Applicable
Intervention
Not specified
Conditions
Anesthesia, Spinal
Sponsor
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Enrollment
51
Locations
1
Primary Endpoint
Number of successful administrations
Status
Completed
Last Updated
3 years ago

Overview

Brief Summary

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.

Registry
clinicaltrials.gov
Start Date
December 16, 2022
End Date
January 10, 2023
Last Updated
3 years ago
Study Type
Observational
Sex
All

Investigators

Eligibility Criteria

Inclusion Criteria

  • A genetically confirmed diagnosis of SMA
  • Treatment with nusinersen

Exclusion Criteria

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

Outcomes

Primary Outcomes

Number of successful administrations

Time Frame: During the procedure

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

Number of attempts

Time Frame: During the procedure

Defined as the number of needle insertions through the skin surface

Procedure time

Time Frame: 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

Time Frame: During the procedure

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

Adverse events (AEs)

Time Frame: 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)

Study Sites (1)

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