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

RecruitingNAAges 3 Years - anyAllAlcyone Therapeutics, Inc
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
Inclusion Criteria: 1. Subject is 3 years or older 2. Subject is diagnosed with SMA and is a candidate for Spinraza treatment as indicated in country-specific 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 country-specific drug labeling 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. Exclusion Criteria: 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, or is a woman of childbearing potential or a man of reproductive age who is unwilling or unable to practice contraception, as described in Section 14.10. 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 (as defined per investigator's discretion) 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 country-specific 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. 14. Subject has clinically meaningful immunosuppression, in the opinion of the investigator, either from therapy or a comorbid condition, which, in the opinion of the investigator, makes them a poor surgical candidate due to increased risk of infection.
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What it tests

  • ThecaFlex DRx System
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Where

21 sites
Barrow Neurological Institute
Phoenix, United States
Children's Hospital Orange County
Orange, United States
Stanford Medical Center
Palo Alto, United States
Rady Children's Hospital
San Diego, United States
Nemours Children's Hospital, Florida
Orlando, United States
Lurie Childrens Hospital
Chicago, United States
Boston Children's Hospital
Boston, United States
Helen DeVos Children's Hospital
Grand Rapids, United States

…and 13 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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