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

Eligibility Criteria

• 1. Subject is 3 years or older
• 2. Subject diagnosed with SMA who are candidates for Spinraza treatment as indicated in its label
• 3. Subject resistant to lumbar puncture (LP), where resistance is defined as:
• 1. 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
• 2. Subjects for whom the treating physician determines implantation of the ThecaFlex DRx™ System is otherwise in the subject's best interest
• 4. 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
• 5. 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
• 6. Subject (or subject's legally authorized representative) is willing and able to provide written informed consent; and
• 7. Subject and parent/caregiver able and willing to complete all clinical investigation procedures, measurements, and visits.
• 1. Subject meets any of the contraindications for use of the ThecaFlex DRx™ System as outlined in the ThecaFlex DRx™ System Instructions for Use
• 2. 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)
• 3. Subject is pregnant or nursing or plans to become pregnant during the course of the clinical investigation
• 4. 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:
• 1. Severe structural impediment that may preclude safe implantation of the catheter and port
• 2. Major medical events within 60 days prior to screening; or
• 3. Relevant surgeries (e.g., head and neck, back, etc.) within 60 days prior to screening or planned during the duration of the clinical investigation
• 4. Space-occupying lesion with mass effect
• 5. Posterior fossa mass
• 6. Arnold-Chiari malformation
• 7. Coagulation abnormalities and/or thrombocytopenia
• 8. Insufficient or inadequate skin or underlying fat to adequately protect the port or enough surface area to allow the skin to close safely
• 5. Subject is contraindicated for administration of nusinersen per its approved labeling
• 6. History of intrathecal granuloma formation
• 7. History of bacterial meningitis or aseptic meningitis within 6 months of screening
• 8. 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
• 9. History of hydrocephalus
• 10. Diagnosed degenerative muscular disease other than SMA
• 11. 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
• 12. Serious medical condition that, in the opinion of the investigator, may lead to reduced life expectancy beyond 12 months.
• 13. Subject is involved in another Investigation Device Exemption Study.