Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study)

Completed

• Conditions: Anesthesia, Spinal; Spinal Puncture Complications; Spinal Muscular Atrophy; Ultrasonography
• Interventions: Procedure: Interlaminar ultrasound-assisted intrathecal administration of nusinersen; Procedure: Landmark-based intrathecal administration of nusinersen

• Registration Number: NCT05644899
• Lead Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-11-30
• Study First Submitted QC: 2022-11-30
• Status Verified: 2022-12
• Study First Posted: 2022-12-09
• Study Start: 2022-12-16
• Primary Completion: 2023-01-10
• Study Completion: 2023-01-10
• Last Update Submitted: 2023-03-07
• Last Update Posted: 2023-03-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 51

Inclusion Criteria

• A genetically confirmed diagnosis of SMA
• Treatment with nusinersen

Exclusion Criteria

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
SMA patients	Interlaminar ultrasound-assisted intrathecal administration of nusinersen	51 adults with a genetically confirmed diagnosis of SMA (13 SMA type 2, 38 SMA type 3) treated with nusinersen. The technique used was based on the anesthesiologist's preference.
SMA patients	Landmark-based intrathecal administration of nusinersen	51 adults with a genetically confirmed diagnosis of SMA (13 SMA type 2, 38 SMA type 3) treated with nusinersen. The technique used was based on the anesthesiologist's preference.

Primary Outcome Measures

Name	Time	Method
Number of successful administrations	During the procedure	Defined as a confirmation of the CSF flow through the spinal needle and subsequent administration of whole drug
Number of attempts	During the procedure	Defined as the number of needle insertions through the skin surface
Procedure time	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	During the procedure	Defined as a successful intrathecal administration with equal or less than 4 attempts
Adverse events (AEs)	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)

Trial Locations

Locations (1)

Fondazione Policlinico Gemelli; 🇮🇹 Roma, RM, Italy