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

Recruiting now · Not applicable

Conditions studied: Spinal Muscular Atrophy, Spine Deformity, Scoliosis

In brief

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.

Key facts

Study ID

NCT05866419

Run by

Alcyone Therapeutics, Inc

People needed

90

Starts

2023-11-27

Expected to finish

2030-07-01

Last updated by the study team

2026-04-16

Who can join

Age: 3 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Subject is 3 years or older
• Subject diagnosed with SMA who are candidates for Spinraza treatment as indicated in its label
• Subject resistant to lumbar puncture (LP), where resistance is defined as:
• Subject with respiratory issues or other comorbidities who is at an increased risk for complications due to the need for repeat anesthesia and imaging radiation exposure to safely perform LP; or
• Subjects for whom the treating physician determines implantation of the ThecaFlex DRx™ System is otherwise in the subject's best interest
• Subject has been prescribed nusinersen via chronic intrathecal bolus administration in accordance with the drug labeling and first port access of the ThecaFlex DRx system is planned within 2 weeks of implantation
• Subject, per Investigator discretion, is able to undergo a percutaneous or open surgical procedure for index port and catheter implantation where at least 7 cm of the ThecaFlex DRx system catheter will be placed intrathecally
• Subject (or subject's legally authorized representative) is willing and able to provide written informed consent; and
• Subject and parent/caregiver able and willing to complete all clinical investigation procedures, measurements, and visits.

You may not qualify if…

• Subject meets any of the contraindications for use of the ThecaFlex DRx™ System as outlined in the ThecaFlex DRx™ System Instructions for Use
• Presence or history of (< 6 months prior to the procedure): an implanted microinfusion pump, intrathecal catheter, or other intrathecal drug delivery devices/component, or implanted shunt for drainage of cerebrospinal fluid (CSF), or reservoir in the CSF space (note: any ThecaFlex Port and Catheter System must not be placed in the same location as a previous indwelling port and catheter)
• Subject is pregnant or nursing or plans to become pregnant during the course of the clinical investigation
• Any condition or event, which in the opinion of the Investigator may adversely affect the safety and effective implantation and use of the ThecaFlex DRx™ system or confound the clinical investigation, including:
• Severe structural impediment that may preclude safe implantation of the catheter and port
• Major medical events within 60 days prior to screening; or
• Relevant surgeries (e.g., head and neck, back, etc.) within 60 days prior to screening or planned during the duration of the clinical investigation
• Space-occupying lesion with mass effect
• Posterior fossa mass
• Arnold-Chiari malformation
• Coagulation abnormalities and/or thrombocytopenia
• Insufficient or inadequate skin or underlying fat to adequately protect the port or enough surface area to allow the skin to close safely
• Subject is contraindicated for administration of nusinersen per its approved labeling
• History of intrathecal granuloma formation
• History of bacterial meningitis or aseptic meningitis within 6 months of screening
• History of tumors or other spinal abnormalities documented by magnetic resonance imaging (MRI) or computed tomography (CT) that would interfere with the catheter implantation procedures or CSF circulation
• History of hydrocephalus
• Diagnosed degenerative muscular disease other than SMA
• History of depression, cognitive impairment, or another psycho-behavioral problem that in the opinion of the Investigator may preclude safe participation in the clinical investigation and
• Serious medical condition that, in the opinion of the investigator, may lead to reduced life expectancy beyond 12 months.
• Subject is involved in another Investigation Device Exemption Study.

Where it is running

• Barrow Neurological Institute — Phoenix, Arizona, United States (enrolling)
• Children's Hospital Orange County — Orange, California, United States (enrolling)
• Stanford Medical Center — Palo Alto, California, United States (enrolling)
• Rady Children's Hospital — San Diego, California, United States (enrolling)
• Nemours Children's Hospital, Florida — Orlando, Florida, United States (enrolling)
• Lurie Childrens Hospital — Chicago, Illinois, United States (enrolling)
• Boston Children's Hospital — Boston, Massachusetts, United States (enrolling)
• Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States (enrolling)
• Columbia University Irving Medical Center/NewYork Presbyterian Hospital — New York, New York, United States (enrolling)
• UH Rainbow Babies and Children's Hospital — Cleveland, Ohio, United States (enrolling)
• Penn State Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States (enrolling)
• Children's Hospital Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Texas Children's Hospital — Houston, Texas, United States (enrolling)
• University of Virginia — Charlottesville, Virginia, United States (enrolling)
• Childrens Hospital of the King's Daughters — Norfolk, Virginia, United States (enrolling)
• Universitätsklinikum Essen — Essen, Germany (enrolling)
• Specialised Hospital Ludwika Rydygiera — Krakow, Poland (enrolling)
• Research Institute of Polish Mother's Memorial Hospital — Lodz, Poland (enrolling)
• Hospital Universitario La Paz — Madrid, Spain (enrolling)
• Hospital Universitario y Politecnico la Fe de Valencia — Valencia, Spain (enrolling)
• Sheffield Teaching Hospitals NHS Foundation Trust — Sheffield, United Kingdom (enrolling)

Full record on ClinicalTrials.gov

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