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New implant could help SMA patients who Can't get spinal injections

NCT ID NCT05866419

Disease control Sponsor: Alcyone Therapeutics, Inc Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 20, 2026 · Updated 2 times

Summary

This trial tests a new implantable device called ThecaFlex DRx in 90 people with spinal muscular atrophy (SMA) who cannot receive standard spinal injections due to spine problems or other risks. The device is placed under the skin and delivers the medication nusinersen directly into the spinal fluid. Researchers will check if the device works safely and effectively over 12 months.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ThecaFlex DRx System (implantable port and catheter)
What this could lead to
If successful, this device could provide a safer, easier way to deliver life-saving SMA medication to patients who currently cannot receive it due to spine problems.
What could go wrong
This is an early feasibility study with no control group, so results may not confirm the device is better than standard care. Implant surgery carries risks like infection or device malfunction.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 90 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2023

Expected to finish

Jul 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

3 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

scoliosis spinal muscular atrophy vertebral column disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Scoliosis Spinal muscular atrophy Spine deformity

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    21 sites in 5 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Barrow Neurological Institute

    RECRUITING

    Phoenix, Arizona, 85013, United States

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Children's Hospital Orange County

    RECRUITING

    Orange, California, 92868, United States

  • Children's Hospital Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Childrens Hospital of the King's Daughters

    RECRUITING

    Norfolk, Virginia, 23510, United States

  • Columbia University Irving Medical Center/NewYork Presbyterian Hospital

    RECRUITING

    New York, New York, 10032, United States

  • Helen DeVos Children's Hospital

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

  • Hospital Universitario La Paz

    RECRUITING

    Madrid, Spain

  • Hospital Universitario y Politecnico la Fe de Valencia

    RECRUITING

    Valencia, 46026, Spain

  • Lurie Childrens Hospital

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Nemours Children's Hospital, Florida

    RECRUITING

    Orlando, Florida, 32827, United States

  • Penn State Milton S. Hershey Medical Center

    RECRUITING

    Hershey, Pennsylvania, 17033, United States

  • Rady Children's Hospital

    RECRUITING

    San Diego, California, 92037, United States

  • Research Institute of Polish Mother's Memorial Hospital

    RECRUITING

    Lodz, Poland

  • Sheffield Teaching Hospitals NHS Foundation Trust

    RECRUITING

    Sheffield, United Kingdom

  • Specialised Hospital Ludwika Rydygiera

    RECRUITING

    Krakow, Poland

  • Stanford Medical Center

    RECRUITING

    Palo Alto, California, 94301, United States

    Contact Email: •••••@•••••

  • Texas Children's Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • UH Rainbow Babies and Children's Hospital

    RECRUITING

    Cleveland, Ohio, 44106, United States

  • University of Virginia

    RECRUITING

    Charlottesville, Virginia, 22903, United States

  • Universitätsklinikum Essen

    RECRUITING

    Essen, Germany

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