Safety and Performance of the ThecaFlex DRx System Port and Catheter for Intrathecal Delivery of Nusinersen in Spinal Muscular Atrophy Patients Resistant to Lumbar Puncture

What this Trial Is About

Researchers are evaluating the clinical use of the ThecaFlex DRx2 System to deliver nusinersen in people aged 3 years and older who have spinal muscular atrophy (SMA) and require chronic intrathecal treatment. This multicenter, multinational study aims to assess the safety and performance of this implantable device designed for patients who are resistant to lumbar puncture or have complex spine conditions. The trial will support a Pre-Market Approval application by collecting data over a 12-month period after implantation. All participants will receive the ThecaFlex DRx2 System, which includes an implantable intrathecal catheter and subcutaneous port to allow repeated bolus administration of nusinersen. Subjects who have previously received nusinersen will have maintenance doses delivered every 4 months through the implant, while those new to nusinersen will receive loading doses at 14-day intervals followed by maintenance doses every 4 months. The study includes a lead-in phase with 10 subjects, followed by expanded enrollment, totaling approximately 90 participants. Participants will be monitored for 12 months after receiving the implant, with evaluations including successful implantation and delivery of therapy. Safety will be carefully reviewed by independent committees throughout the study. Assessments will track device-related events, procedural complications, anesthesia and radiation exposure, and nusinersen-related side effects. The total study duration for each participant is expected to be about 37 months, with overall trial completion around 60 months.

Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy