PIERRE

• Research type

  Research Study

• Full title

  Safety and Performance of the ThecaFlex DRx™ System Port and Catheter for Chronic Intrathecal Access, Cerebrospinal Fluid (CSF) Aspiration, and DElivery of Nusinersen in Spinal Muscular Atrophy (SMA) Patients Resistant to Lumbar PunctuRE (PIERRE) Trial

• IRAS ID

  337123

• Contact name

  Channa Hewamadduma

• Contact email

  Chewamadduma1@sheffield.ac.uk

• Sponsor organisation

  Alcyone Therapeutics, Inc.

• Clinicaltrials.gov Identifier

  NCT05866419

• Duration of Study in the UK

  3 years, 10 months, 31 days

• Research summary

  The clinical investigation aims to evaluate the safety and performance of the ThecaFlex DRx™ System. The primary goal is to demonstrate its successful use in delivering nusinersen to patients with spinal muscular atrophy (SMA). A key secondary objective is to determine if the ThecaFlex DRx™ System reduces the need for anesthesia and radiation exposure compared to repeated lumbar punctures for nusinersen infusion. Additionally, the investigation will assess the safety of the ThecaFlex DRx™ System in this context. All enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRx™ System) and will be
  followed for 12 months after receiving the implant. The 12-month data will be used to assess the primary endpoint
  support a Pre-Market Approval (PMA) application.

• REC name

  South Central - Berkshire Research Ethics Committee

• REC reference

  25/SC/0160

• Date of REC Opinion

  27 May 2025

• REC opinion

  Unfavourable Opinion