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New implant could change how SMA patients get their medicine

NCT ID NCT06555419

Disease control Sponsor: Biogen 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 04, 2026 · Updated 4 times

Summary

This study looks at how the body handles the drug nusinersen when given through a new implantable device called ThecaFlex DRx™, compared to the usual method of a lumbar puncture (spinal tap). About 58 people with spinal muscular atrophy (SMA) who are already in another study (PIERRE) will receive one dose by lumbar puncture and one dose through the device. Researchers will measure drug levels in the blood to see if the device works as well as the standard procedure.

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

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 78 people

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

Started

Jan 2025

Expected to finish

Sep 2027

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.

Key Inclusion Criteria: \- Participants must be screened in the PIERRE study to be eligible for enrollment in the PIERRE PK study. Participants in the 12 mg group * Participant must have completed all 4 required 12 mg loading doses as per the approved 12 mg dose regimen. * Participant is on regular LP maintenance dosing of nusinersen 12 mg every 4 months, with 120 days ± 14 days between the LP-delivered study dose and the last nusinersen dose prior to study enrollment. Participants in the 28 mg group * Participant must have completed 1 or 2 of the 50 mg loading doses as per the approved 50/28 mg dose regimen. * Participant is on regular LP maintenance dosing of nusinersen 28 mg every 4 months, with 120 days ± 14 days between the LP-delivered study dose and the last nusinersen dose prior to study enrollment. Key Exclusion Criteria: * Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrollment in other interventional clinical trials for the treatment of SMA (except for the PIERRE study or interventional clinical trials of myostatin inhibitors \[e.g., apitegromab and taldefgrobep alfa\]). * Participant is naïve to nusinersen treatment. * Participant is receiving nusinersen at a dose other than 12 mg or 28 mg. * Participant has already undergone implantation of the ThecaFlex DRx system or any other implantable medical device for intrathecal (IT) administration. * Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study. NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

spinal muscular atrophy

As listed by the trial registrant

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

Muscular atrophy, spinal

How to take part

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

  1. The places running it

    19 sites in 6 countries. The list below names each one and where it is.

  2. 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 ↗

  3. 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

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611-2605, United States

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Children's Hospital of Orange County

    RECRUITING

    Orange, California, 92868, United States

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of the King's Daughters_Norfolk

    RECRUITING

    Norfolk, Virginia, 23507, United States

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    WITHDRAWN

    Roma, 168, Italy

  • Fondazione Serena Onlus - Centro Clinico Nemo_Milano

    WITHDRAWN

    Milan, 20162, Italy

  • Helen DeVos Children's Hospita

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

  • Hospital Universitari i Politecnic La Fe_Valencia

    RECRUITING

    Valencia, 46026, Spain

  • Hospital Universitario La Paz

    RECRUITING

    Madrid, 28046, Spain

  • Hôpital Raymond Poincaré

    RECRUITING

    Garches, Hauts De Seine, 92380, France

  • Instytut Centrum Zdrowia Matki Polki

    RECRUITING

    Lodz, 93-338, Poland

  • Milton S. Hershey Medical Center | Pennsylvania State University_Hershey

    RECRUITING

    Hershey, Pennsylvania, 17033, United States

  • Nemours Children's Hospital, Orlando

    RECRUITING

    Orlando, Florida, 32827, United States

  • Royal Hallamshire Hospital Neurology Department

    RECRUITING

    Sheffield, South Yorkshire, S102JF, United Kingdom

  • St. Joseph's Hospital and Medical Center - Barrow Neurological Institute

    RECRUITING

    Phoenix, Arizona, 85013, United States

  • Stacey Hall Developmental Pediatrics

    RECRUITING

    Charlottesville, Virginia, 22903, United States

  • Stanford University Medical Center | Department of Neurology_Palo Alto

    RECRUITING

    Palo Alto, California, 94304, United States

  • Szpital Specjalistyczny im. L.Rydygiera w Krakowie

    RECRUITING

    Krakow, 31-826, Poland

  • Texas Childrens Hospital Houston

    RECRUITING

    Houston, Texas, 77030, United States

  • Universitaetsklinikum Essen

    RECRUITING

    Essen, 45147, Germany

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