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

August 18, 2026 updated by: Alcyone Therapeutics, Inc

Safety and Performance of the ThecaFlex DRx™ System Port and Catheter for Chronic Intrathecal Access, Cerebrospinal Fluid (CSF) Aspiration, and DElivery of Nusinersen in Spinal Muscular Atrophy (SMA) Patients Resistant to Lumbar PunctuRE Trial (PIERRE)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

90

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Germany
  • Poland
  • Spain
  • United Kingdom
      • Sheffield, United Kingdom
        • Recruiting
        • Sheffield Teaching Hospitals NHS Foundation Trust
        • Contact:
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
    • California
      • Orange, California, United States, 92868
      • Palo Alto, California, United States, 94301
      • San Diego, California, United States, 92037
    • Florida
      • Orlando, Florida, United States, 32827
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Massachusetts
    • Michigan
    • New York
      • New York, New York, United States, 10032
        • Recruiting
        • Columbia University Irving Medical Center/NewYork Presbyterian Hospital
        • Contact:
    • Ohio
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital Philadelphia
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
    • Virginia
      • Charlottesville, Virginia, United States, 22903
      • Norfolk, Virginia, United States, 23510

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The proportion of subjects with successful Implantation of the ThecaFlex DRx™ Port and Catheter
Time Frame: 12 Months
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).
12 Months
The proportion of subjects with successful Intrathecal Therapy Infusion(s) through 12 months
Time Frame: 12 Months
12 Months

Secondary Outcome Measures

Outcome Measure
Time Frame
Proportion of subjects with reduced anesthesia and radiation exposure compared to repeat LP for nusinersen infusion in SMA subjects.
Time Frame: 12 months
12 months
Incidence of device-related adverse events (AEs)/complications through 12 months
Time Frame: 12 months
12 months
Incidence of procedural complications through 12 months
Time Frame: 12 months
12 months
Incidence of nusinersen-related AEs
Time Frame: 12 months
12 months
Mean duration of radiation exposure from implant through 12-month follow-up
Time Frame: 12 months
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alcyone Therapeutics, Inc

Collaborators

Biogen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 27, 2023

Primary Completion (Estimated)

April 1, 2028

Study Completion (Estimated)

July 1, 2030

Study Registration Dates

First Submitted

May 10, 2023

First Submitted That Met QC Criteria

May 10, 2023

First Posted (Actual)

May 19, 2023

Study Record Updates

Last Update Posted (Actual)

August 19, 2026

Last Update Submitted That Met QC Criteria

August 18, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • QT-0179

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

Yes

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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