Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy (NCT05866419)
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 Trial (PIERRE)
🩺 Plain-English Summary (8th-Grade Level)
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA). 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.
Who can join: Review the eligibility criteria below.
Location: Barrow Neurological Institute — Phoenix, Arizona
Age range: 3 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (21 Sites)
- Barrow Neurological Institute — Phoenix, Arizona
- Children's Hospital Orange County — Orange, California
- Stanford Medical Center — Palo Alto, California
- Rady Children's Hospital — San Diego, California
- Nemours Children's Hospital, Florida — Orlando, Florida
- Lurie Childrens Hospital — Chicago, Illinois
- Boston Children's Hospital — Boston, Massachusetts
- Helen DeVos Children's Hospital — Grand Rapids, Michigan
- Columbia University Irving Medical Center/NewYork Presbyterian Hospital — New York, New York
- UH Rainbow Babies and Children's Hospital — Cleveland, Ohio
- Penn State Milton S. Hershey Medical Center — Hershey, Pennsylvania
- Children's Hospital Philadelphia — Philadelphia, Pennsylvania
- Texas Children's Hospital — Houston, Texas
- University of Virginia — Charlottesville, Virginia
- Childrens Hospital of the King's Daughters — Norfolk, Virginia
- Universitätsklinikum Essen — Essen,
- Specialised Hospital Ludwika Rydygiera — Krakow,
- Research Institute of Polish Mother's Memorial Hospital — Lodz,
- Hospital Universitario La Paz — Madrid,
- Hospital Universitario y Politecnico la Fe de Valencia — Valencia,
- Sheffield Teaching Hospitals NHS Foundation Trust — Sheffield,
📞 Contact the Study Coordinator
Phone: (857) 500-7526
Email: janelle.arrambide@biogen.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Spinal Muscular Atrophy
- Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) — Phoenix, Arizona
- Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb — Pittsburgh, Pennsylvania
- A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) — Phoenix, Arizona
- Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab — Phoenix, Arizona
- Spinraza in Adult Spinal Muscular Atrophy — Phoenix, Arizona
- Early Check: Expanded Screening in Newborns — Research Triangle Park, North Carolina
Explore by condition: All Spinal Muscular Atrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.