Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy (NCT05866419) | Clinical Trial Compass
RecruitingNot Applicable
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
United States90 participantsStarted 2023-11-27
Plain-language summary
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRxâ„¢ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).
All enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRxâ„¢ 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 participate
Age range3 Years
SexALL
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Inclusion criteria
✓. 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
Exclusion criteria
✕. 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)
What they're measuring
1
The proportion of subjects with successful Implantation of the ThecaFlex DRxâ„¢ Port and Catheter
Timeframe: 12 Months
2
The proportion of subjects with successful Intrathecal Therapy Infusion(s) through 12 months
✕. 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