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Led by Alcyone Therapeutics, Inc · Updated on 2026-04-16
90
Participants Needed
21
Research Sites
117 weeks
Total Duration
Researchers are evaluating the clinical use of the ThecaFlex DRx2 System to deliver nusinersen in people aged 3 years and older who have spinal muscular atrophy SMA and require chronic intrathecal treatment. This multicenter, multinational study aims to assess the safety and performance of this implantable device designed for patients who are resistant to lumbar puncture or have complex spine conditions. The trial will support a Pre-Market Approval application by collecting data over a 12-month period after implantation. All participants will receive the ThecaFlex DRx2 System, which includes an implantable intrathecal catheter and subcutaneous port to allow repeated bolus administration of nusinersen. Subjects who have previously received nusinersen will have maintenance doses delivered every 4 months through the implant, while those new to nusinersen will receive loading doses at 14-day intervals followed by maintenance doses every 4 months. The study includes a lead-in phase with 10 subjects, followed by expanded enrollment, totaling approximately 90 participants. Participants will be monitored for 12 months after receiving the implant, with evaluations including successful implantation and delivery of therapy. Safety will be carefully reviewed by independent committees throughout the study. Assessments will track device-related events, procedural complications, anesthesia and radiation exposure, and nusinersen-related side effects. The total study duration for each participant is expected to be about 37 months, with overall trial completion around 60 months.
CONDITIONS
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
J
Janelle Arrambide
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Alcyone Therapeutics, Inc
Lead Sponsor
Biogen
Collaborating Sponsor
This table compares participation in this trial with other options available for this condition. The information is drawn from published research and the trial’s study details and is provided for educational purposes only.
| Factors | Implantable ThecaFlex DRx Port for Nusinersen Delivery | Repeated Lumbar Puncture for Nusinersen Delivery |
|---|---|---|
| What is it? | An implantable port and intrathecal catheter system designed for ongoing delivery of nusinersen directly into the cerebrospinal fluid in people with SMA, providing an alternative for those unable to tolerate or undergo repeated lumbar punctures. | The standard method for administering nusinersen in SMA involves repeated lumbar punctures, which allow direct delivery of the medication into the cerebrospinal fluid. |
| Role in study or care | Served as the investigational method for delivering intrathecal nusinersen to people with SMA who are poor candidates for or resistant to standard lumbar puncture. | Functions as the ongoing standard practice for intrathecal administration of nusinersen in eligible SMA patients. |
| Primary use | Used for long-term, repeated delivery of nusinersen in patients with SMA who are unable or unsuitable to receive the medication by lumbar puncture due to spine abnormalities or procedural risk. | Employed to provide direct cerebrospinal fluid access for nusinersen administration in SMA patients who do not have barriers to repeated lumbar punctures. |
| How it is performed or given | A surgical procedure is used to implant the ThecaFlex DRx catheter into the intrathecal space, with a subcutaneous port placed for ongoing bolus access; medication is then administered via the port during scheduled treatments. | A needle is inserted between lower back vertebrae at each scheduled timepoint to access the cerebrospinal fluid and administer nusinersen, typically under local anesthesia or sedation. |
| Evidence status | Preliminary clinical results from pilot studies and registry indicate feasibility for repeated nusinersen delivery in SMA patients with difficult lumbar access, but large-scale or long-term outcome evidence is still emerging. | Supported by extensive clinical evidence as the routine route for administering nusinersen effectively and safely in eligible SMA patients. |
| Important limitations | Requires surgical implantation, which carries associated operative and post-operative risks. Long-term safety, device complications, and outcomes are not yet established in large studies. | Repeated lumbar punctures may not be possible in patients with severe scoliosis or other spinal deformities, and can increase exposure to anesthesia and imaging radiation, particularly in children and those needing frequent treatments. |
Disclaimer: This comparison is created with the help of artificial intelligence(AI) and may contain errors or missing information. It is not medical advice and does not mean one option is better or safer than another. Clinical trials are still being studied, and joining a trial is your choice. Please consult your healthcare professional before making any decisions.
You may qualify if you...
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Duration - Up to 200 days
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - 1 day
Participants undergo implantation of the ThecaFlex DRx™ System port and catheter device for intrathecal delivery of nusinersen.
1 visit (in-person)
Duration - 12 months
Participants receive intrathecal nusinersen dosing via the implanted ThecaFlex DRx System according to their treatment history, with naïve participants receiving loading doses followed by maintenance doses and non-naïve participants receiving maintenance doses every 4 months.
Multiple dosing visits over 12 months, including doses at 14-day intervals for initial loading (if naïve) and maintenance doses every 4 months
Duration - 12 months
Participants are monitored for safety and device performance for 12 months following implantation.
Regular safety and performance monitoring visits over 12 months
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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Ignacio Mesina-Estarrón, Michael Abruzzo, Fernando Cotrim Gomes...
https://pubmed.ncbi.nlm.nih.gov/41830093/Alcyone Therapeutics, Inc
Lead Sponsor
Biogen
Collaborating Sponsor
