Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age3+
SponsorAlcyone Therapeutics, Inc
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Subject is 3 years or older
Subject is diagnosed with SMA and is a candidate for Spinraza treatment as indicated in country-specific label
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
Disqualifiers
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, 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.
Severe structural impediment that may preclude safe implantation of the catheter and port
Trial design
Design model
Single group
Treatments tested in this trial
ThecaFlex DRx System
DeviceThe 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.
Treatment groups
90 Participants
are divided into 1 treatment groupGroup A: ThecaFlex DRx Port and Catheter SystemExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
The proportion of subjects with successful Implantation of the ThecaFlex DRx™ Port and Catheter
The primary outcome measures the proportion of subjects with successful implantation of the ThecaFlex DRx™ Port and Catheter System who are able to receive successful intrathecal therapy infusion(s).
Time frame
12 Months2
The proportion of subjects with successful Intrathecal Therapy Infusion(s) through 12 months
Time frame
12 MonthsSecondary outcomes
1
Proportion of subjects with reduced anesthesia and radiation exposure compared to repeat LP for nusinersen infusion in SMA subjects.
Time frame
12 months2
Incidence of device-related adverse events (AEs)/complications through 12 months
Time frame
12 months3
Incidence of procedural complications through 12 months
Time frame
12 months4
Incidence of nusinersen-related AEs
Time frame
12 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Alcyone Therapeutics, Inc
Lead sponsor
Biogen
Collaborator
Source ClinicalTrials.gov