ClinicalTrials.gg
RecruitingNCT05866419PIERREUpdated Nov 9, 2026

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

An interventional study of ThecaFlex DRx System in Spinal Muscular Atrophy, Spine Deformity and Scoliosis, sponsored by Alcyone Therapeutics, Inc. Recruiting at 23 sites in 5 countries. Open to participants aged 3 Years and older. Per ClinicalTrials.gov, last updated 2026-11-09.

Sponsored by Alcyone Therapeutics, Inc · Not applicable, Interventional, and Treatment

From the registry’s dates

  • Started Nov 2023; still recruiting 2 years 11 months later.
Updated Nov 9, 20262 sites addedGo to Updates ↓
Phase
Not applicable
Study type
Interventional
Enrollment
90
Allocation
Not applicable
Ages
3 Years and older
Sex
All
01

Study summary

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.

Read the detailed description

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.

02

Conditions studied

  • Spinal Muscular Atrophy
  • Spine Deformity
  • Scoliosis
03

In context

Muscular Atrophy, Spinal

282 studies on the registry are indexed under Muscular Atrophy, Spinal; 73 are open to participants now.

This study's planned enrollment of 90 is above the median of 29 across 156 interventional studies indexed under Muscular Atrophy, Spinal.

Browse Muscular Atrophy, Spinal studies →

Lead sponsor

Alcyone Therapeutics, Inc is the lead sponsor of 2 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
3 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 ; and
  7. Subject and parent/caregiver able and willing to complete all clinical investigation procedures, measurements, and visits.

Exclusion criteria

Exclusion Criteria:

  1. Subject meets any of the 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 or a man of reproductive age who is unwilling or unable to practice , 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 beyond 12 months.
  13. Subject is involved in another Investigation Device Exemption Study.
  14. Subject has , 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.
Terms in these criteria, in plain language(7)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Contraindication

A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Life expectancy

The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Immunosuppression

A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.

Read whether the line is about a condition or about medicines, and which medicines and doses it means.

05

Study design

Phase
Not applicable
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
90 participants (estimated)

Study arms

  • Experimental
    ThecaFlex DRx Port and Catheter System

    Subjects who meet all of the inclusion and none of the exclusion would be eligible to receive the ThecaFlex DRx Port and Catheter System.

    Device: ThecaFlex DRx System

Interventions

  • DeviceThecaFlex DRx System

    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.

06

What researchers measure

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 Months

  2. The proportion of subjects with successful Intrathecal Therapy Infusion(s) through 12 months

    Time frame: 12 Months

Secondary outcomes

  1. Proportion of subjects with reduced anesthesia and radiation exposure compared to repeat LP for nusinersen infusion in SMA subjects.

    Time frame: 12 months

  2. Incidence of device-related adverse events (AEs)/complications through 12 months

    Time frame: 12 months

  3. Incidence of procedural complications through 12 months

    Time frame: 12 months

  4. Incidence of nusinersen-related AEs

    Time frame: 12 months

  5. Mean duration of radiation exposure from implant through 12-month follow-up

    Time frame: 12 months

07

Study locations

21 of 23 sites recruiting
08

References and documents

Publications

  • Mesina-Estarron I, Abruzzo M, Gomes FC, Thaxton M, Yengo-Kahn A, Stone S, Northam W, Snyder B, Cahill P, Bauer D, Muhonen M. Clinical evaluation of ThecaFlex DRx, a novel implantable catheter-port for intrathecal nusinersen delivery in spinal muscular atrophy: Initial results from the PIERRE-IDE study. J Neuromuscul Dis. 2026 Mar 13:22143602261432303. doi: 10.1177/22143602261432303. Online ahead of print. PubMed 41830093 ↗

Individual participant data

Plan to share: No

09

Updates

1 registry update since Sep 25, 2026
Sites
2 sites added
Show 2 added (2 United States)
  • John's Hopkins Children's Center · Baltimore, United States
  • Dell Children's Medical Center of Central Texas · Austin, United States
Nov 9, 2026
Site contacts
Changed at 2 sites. See the sites
Show 2 sites
  • Penn State Milton S. Hershey Medical Center · Hershey, United StatesContact a•••@pennstatehealth.psu.edu→k•••@pennstatehealth.psu.edu
  • Childrens Hospital of the King's Daughters · Norfolk, United StatesContact E•••@chkd.org→p•••@chkd.org
Nov 9, 2026
Show all 1 update
  1. Nov 9, 2026
    2 sites added
    Show 2 added (2 United States)
    • John's Hopkins Children's Center · Baltimore, United States
    • Dell Children's Medical Center of Central Texas · Austin, United States
    Site contacts Changed at 2 sites. See the sites
    Show 2 sites
    • Penn State Milton S. Hershey Medical Center · Hershey, United StatesContact a•••@pennstatehealth.psu.edu→k•••@pennstatehealth.psu.edu
    • Childrens Hospital of the King's Daughters · Norfolk, United StatesContact E•••@chkd.org→p•••@chkd.org
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT05866419
Lead sponsor
Alcyone Therapeutics, Inc
Collaborators
Biogen
Responsible party
Sponsor
First posted
May 19, 2023
Start date
Nov 27, 2023
Primary completion
Apr 2028 (estimated)
Completion
Jul 2030 (estimated)
Last update
Nov 9, 2026

Study contacts

Janelle Arrambide
Contact
janelle.arrambide@biogen.com
857-500-7526

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
Yes
View the source record on ClinicalTrials.gov ↗

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