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)
Alcyone Therapeutics, Inc
Summary
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.
Description
This is a multicenter, multi-national, prospective, non-randomized, single arm, group sequential design, pivotal clinical investigation (device exemption study) to assess the safety and performance of the ThecaFlex DRx™ System in subjects with SMA. The subject population will include subjects aged ≥ 3 years with SMA who are considered candidates for intrathecal port and catheter implantation because they require chronic, bolus intrathecal administration of necessary therapy, and who meet all the inclusion/exclusion criteria for the clinical investigation. After a screening period of up to 20…
Eligibility
- Age range
- 3+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion 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 prescr…
Interventions
- DeviceThecaFlex DRx System
The ThecaFlex DRx™ System consists of an implantable intrathecal catheter, and an implantable subcutaneous port, designed specifically to meet the implantation, Cerebrospinal Fluid (CSF) aspiration, and intrathecal delivery and dosing needs of patients (aged ≥ 3 years) requiring repeated/on-going intermittent chronic bolus intrathecal treatment for life threatening, debilitating Central Nervous System (CNS) disorders who are precluded from Lumbar Puncture (LP) due to complex spine conditions, or who are resistant to LP for therapy administration or CSF aspiration.
Locations (21)
- Barrow Neurological InstitutePhoenix, Arizona
- Children's Hospital Orange CountyOrange, California
- Stanford Medical CenterPalo Alto, California
- Rady Children's HospitalSan Diego, California
- Nemours Children's Hospital, FloridaOrlando, Florida
- Lurie Childrens HospitalChicago, Illinois