Skip to main content
UK clinical trials - updated daily from ClinicalTrials.gov
TrialConnect
← Back to Search
Looking for participantsNA

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

Sponsor: Alcyone Therapeutics, Inc

NCT ID: NCT05866419

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
ThecaFlex DRx System (device)
How long the study runs
Study runs about 80 months (dates as stated)
About the drug or intervention
ThecaFlex DRx System — device: 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.
Patient visit burden
Not specified by the sponsor

In plain English

This study looks at a device called the ThecaFlex DRx System, which is a port (a small device placed under the skin) and a thin tube, for people with spinal muscular atrophy (SMA), a condition that weakens muscles, sometimes alongside spine deformity or scoliosis (a sideways curve of the spine). It is for people aged 3 and over who take a medicine called nusinersen (Spinraza) and for whom a lumbar puncture (a needle into the lower back) is difficult or risky. The sponsor is Alcyone Therapeutics, Inc.

Who can take part

  • Aged 3 years or older
  • Diagnosed with spinal muscular atrophy (SMA) and able to have Spinraza treatment under their country's guidelines
  • Lumbar puncture (a needle into the lower back to give medicine) is difficult or risky for them, for example due to breathing problems or other health conditions, or their doctor believes this device is in their best interest
  • Already prescribed nusinersen given into the lower back by regular injections
  • Able to have a surgical procedure to place the port and tube, with at least 7 cm of the tube placed in the fluid around the spinal cord
  • Able to give written informed consent (or a legal representative can)
  • Able and willing, with a parent or carer, to complete all study visits, checks and procedures

Who may not be able to

  • Any reason the ThecaFlex DRx System instructions say it should not be used
  • Has or recently had (within 6 months) certain devices in the body, such as a medicine pump, a tube in the spinal fluid, a shunt draining spinal fluid, or a reservoir in the spinal fluid space
  • Pregnant, breastfeeding, planning pregnancy, or unable/unwilling to use contraception if of childbearing age
  • Any condition the doctor thinks could make the device unsafe or affect the study, such as a severe blockage in the spine, a major medical event in the last 60 days, spine or head surgery in the last 60 days or surgery planned during the study, certain growths or malformations in the brain or spine, blood clotting problems, or not enough skin or fat to protect the port
  • Cannot have nusinersen according to its approved labelling
  • Past growths (granulomas) caused by tubes in the spinal fluid
  • Meningitis (infection or inflammation of the lining of the brain) within the last 6 months
  • Spine problems seen on scans that would block placing the tube or the flow of spinal fluid
  • History of hydrocephalus (fluid on the brain)
  • Another muscle-wasting disease besides SMA
  • Depression, thinking problems or other mental health problems that the doctor thinks could make taking part unsafe
  • A serious illness that the doctor thinks may shorten life to under 12 months
  • Taking part in another study of an investigational device
  • A weakened immune system that the doctor thinks makes surgery too risky due to infection risk

What taking part involves

  • • Surgery to place the ThecaFlex DRx System under the skin, with at least 7 cm of its tube placed into the fluid around the spinal cord
  • • The system is designed to deliver nusinersen (Spinraza) into the spinal fluid instead of repeated lumbar punctures

Time commitment: Not stated — ask the trial team; taking part involves completing all study visits, checks and procedures, and the surgical procedure to implant the port and tube.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
3 Years and over
Who
All
Number of participants
90
Started
2023-11-27
Last checked
2026-08

Plain English Summary

What is this study?

  • • Testing a new treatment for spinal muscular atrophy
  • • NA - 90 participants
  • • 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)

Who can take part?

  • • Ages 3 Years and over
  • • Diagnosed with spinal muscular atrophy

Where?

  • • Sheffield - Sheffield Teaching Hospitals NHS Foundation Trust

This is a simplified summary. Always discuss with your doctor before making any decisions.

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.

More detail

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 200 days, all enrolled subjects will undergo implantation of the investigational device (ThecaFlex DRx™ System) and will be followed for 12 months after receiving the implant. In agreement with the nusinersen dosing information, the treatment frequency will be adapted for non-naïve and naïve subjects: Subjects who are not naïve to nusinersen should have nusinersen maintenance doses administered via the implant every 4 months (i.e., 4, 8, and 12 months after their last nusinersen dose, before enrollment in the clinical investigation). Subjects who are naïve to nusinersen will have their loading and maintenance doses administered via the device according to the drug labeling. A DMC and Clinical Events Committee (CEC), independent of each other and the Sponsor, will be used to perform ongoing consistent adjudication of events related to the safety throughout the entire duration of the clinical investigation and to periodically review data that relate to the safety of the study. The clinical investigation will enroll 90 subjects. The clinical investigation incorporates a lead-in phase which consists of 10 subjects enrolled, implanted, and followed for 30-days. Enrollment for the lead-in phase is expected to take approximately 6 to 9 months, at which point the Data Monitoring Committee (DMC) will review the data for safety and approve continuation of enrollment. The second phase of enrollment is expected to take approximately 12 to 15 months. Individual subjects are anticipated to be enrolled in the clinical investigation for approximately 37 months. The total duration of this clinical investigation is estimated to be approximately 60 months.

Spinal Muscular AtrophySpine DeformityScoliosis

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 3 Years and over
  • Who can join: All genders

What the study is looking for

  • ✓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 resistant to lumbar puncture (LP), where resistance is defined as:
  • ✓Subject with respiratory issues or other other health conditions who is at an increased risk for complications due to the need...
  • ✓Subjects for whom the treating physician determines implantation of the ThecaFlex DRx™ System is otherwise in the...

Who cannot take part

  • ✗Subject meets any of the contraindications for use of the ThecaFlex DRx System as outlined in the ThecaFlex DRx...
  • ✗Severe structural impediment that may preclude safe implantation of the catheter and port
  • ✗Major medical events (as defined per investigator's discretion) 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...
  • ✗Space-occupying lesion with mass effect
See the full criteria
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.

Where Is This Study? (1 UK site)

Sheffield Teaching Hospitals NHS Foundation Trust

Sheffield, United Kingdom

Recruiting
Site contact (verified)

How to Get in Touch

Janelle Arrambide

Sponsor contact

CONTACT

857-500-7526 janelle.arrambide@biogen.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-08