A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK) (PIERRE-PK)

September 2, 2026 updated by: Biogen

An Open Label Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)

In this PIERRE-PK study, researchers will learn how the body processes nusinersen when it is given through the ThecaFlex DRx™ System, compared to when nusinersen is given by lumbar puncture (LP). The ThecaFlex DRx system is an investigational implantable medical device developed by Alcyone Therapeutics, Inc. It consists of a catheter, which is a flexible tube, connected to a port which is placed under the skin. Alcyone Therapeutics, Inc. has an ongoing study called PIERRE to test the ThecaFlex DRx system. Participants with spinal muscular atrophy (SMA) in the PIERRE study may be enrolled in the PIERRE-PK study.

The main goal of the PIERRE-PK study is to learn how the body processes nusinersen when given by the ThecaFlex DRx system compared to a lumbar puncture. The main questions researchers want to answer are:

  • What is the highest amount of nusinersen found in the blood after dosing?
  • How much nusinersen is found in the blood over the first 24 hours after dosing?

The PIERRE-PK study will be done as follows:

  • Participants will be screened to check if they can join the study. The screening period will be up to 30 days for this study and may overlap with the PIERRE study.
  • Participants will join 1 of 2 groups based on their ongoing nusinersen treatment. One group will be receiving 12 mg doses of nusinersen. The other group will be receiving 28 mg doses of nusinersen.
  • Participants will first receive a dose of nusinersen by lumbar puncture.
  • The ThecaFlex DRx system will be implanted after the lumbar puncture, as part of the PIERRE study.
  • Participants will receive a dose of nusinersen by the ThecaFlex DRx system, as part of the PIERRE study. This dose will be a regular maintenance dose that happens about 4 months after their first dose in this study.
  • Researchers will take blood samples before and after each dose. The last blood sample will be taken 24 hours after the dose.
  • The total study duration for each participant in the PIERRE-PK study will be approximately 5 months. This period will overlap with the participant's first 5 months in the PIERRE study.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

The primary objective of this study is to assess the Pharmacokinetic (PK) profile of nusinersen delivered via standard LP and via the ThecaFlex DRx System in participants with SMA.

Study Type

Interventional

Enrollment (Estimated)

78

Phase

  • Phase 1

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 Contact Backup

Study Locations

  • France
    • Hauts De Seine
      • Garches, Hauts De Seine, France, 92380
        • Recruiting
        • Hôpital Raymond Poincaré
        • Contact:
          • Phone Number: +33147104684
        • Principal Investigator:
          • Marta Gomez
  • Germany
      • Essen, Germany, 45147
        • Recruiting
        • Universitaetsklinikum Essen
        • Principal Investigator:
          • Tim Hagenacker
        • Contact:
          • Phone Number: 49 201 7230
  • Italy
      • Milan, Italy, 20162
        • Withdrawn
        • Fondazione Serena Onlus - Centro Clinico Nemo_Milano
      • Roma, Italy, 168
        • Withdrawn
        • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
  • Poland
      • Krakow, Poland, 31-826
        • Recruiting
        • Szpital Specjalistyczny im. L.Rydygiera w Krakowie
        • Principal Investigator:
          • Ryszard Nowak
        • Contact:
          • Phone Number: 48604565621
      • Lodz, Poland, 93-338
        • Recruiting
        • Instytut Centrum Zdrowia Matki Polki
        • Contact:
          • Phone Number: +48606989185
        • Principal Investigator:
          • Przyslo Lukasz
  • Spain
      • Madrid, Spain, 28046
        • Recruiting
        • Hospital Universitario La Paz
        • Contact:
          • Phone Number: +34917277388
        • Principal Investigator:
          • Maria Garcia Romero
      • Valencia, Spain, 46026
        • Recruiting
        • Hospital Universitari i Politecnic La Fe_Valencia
        • Principal Investigator:
          • Juan Francisco Vazquez Costa
        • Contact:
          • Phone Number: 34 961 24 55 42
  • United Kingdom
    • South Yorkshire
      • Sheffield, South Yorkshire, United Kingdom, S102JF
        • Recruiting
        • Royal Hallamshire Hospital Neurology Department
        • Contact:
          • Phone Number: +441142713708
        • Principal Investigator:
          • Chanma Hewadduma
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Recruiting
        • St. Joseph's Hospital and Medical Center - Barrow Neurological Institute
        • Contact:
          • Phone Number: 602-406-6262
        • Principal Investigator:
          • Shafeeq Ladha
    • California
      • Orange, California, United States, 92868
        • Recruiting
        • Children's Hospital of Orange County
        • Contact:
          • Phone Number: 714-509-3605
        • Principal Investigator:
          • Julian Thomas
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Stanford University Medical Center | Department of Neurology_Palo Alto
        • Principal Investigator:
          • John Day
        • Contact:
          • Phone Number: 650-723-5152
    • Florida
      • Orlando, Florida, United States, 32827
        • Recruiting
        • Nemours Children's Hospital, Orlando
        • Contact:
          • Phone Number: 407-650-7715
        • Principal Investigator:
          • Abdul Hamid Omer
    • Illinois
      • Chicago, Illinois, United States, 60611-2605
        • Recruiting
        • Ann & Robert H. Lurie Children's Hospital of Chicago
        • Contact:
          • Phone Number: 312-227-3550
        • Principal Investigator:
          • Nancy Kuntz
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Boston Children's Hospital
        • Contact:
          • Phone Number: 617-355-8036
        • Principal Investigator:
          • Basil T Darras
    • Michigan
      • Grand Rapids, Michigan, United States, 49503
        • Recruiting
        • Helen DeVos Children's Hospita
        • Contact:
          • Phone Number: 616-391-2241
        • Principal Investigator:
          • Jena Krueger
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Recruiting
        • Milton S. Hershey Medical Center | Pennsylvania State University_Hershey
        • Contact:
          • Phone Number: 717-232-5443
        • Principal Investigator:
          • Dustin Paul
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Contact:
          • Phone Number: 215-590-3174
        • Principal Investigator:
          • Patrick Cahill
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Texas Childrens Hospital Houston
        • Contact:
          • Phone Number: 832-822-1750
        • Principal Investigator:
          • David Bauer
    • Virginia
      • Charlottesville, Virginia, United States, 22903
        • Recruiting
        • Stacey Hall Developmental Pediatrics
        • Contact:
          • Phone Number: 434-924-8184
        • Principal Investigator:
          • Rebecca J Scharf
      • Norfolk, Virginia, United States, 23507
        • Recruiting
        • Children's Hospital of the King's Daughters_Norfolk
        • Principal Investigator:
          • Crystal Proud
        • Contact:
          • Phone Number: 757-668-7000

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

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.

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: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Nusinersen 12 mg Via LP and ThecaFlex DRx System
Participants will receive a maintenance dose of nusinersen, 12 milligrams (mg) via LP in the PIERRE-PK study, followed by implantation of the ThecaFlex DRx System and the subsequent nusinersen 12 mg maintenance dose via the system in the PIERRE study.
Drug: Nusinersen
Administered as specified in the treatment arm.
Other Names:
  • BIIB058
Device: ThecaFlex DRx System
Implanted as specified in the treatment arm.
Experimental: Nusinersen 28 mg Via LP and ThecaFlex DRx System
Participants will receive a maintenance dose of nusinersen, 28 mg via LP in the PIERRE-PK study, followed by implantation of the ThecaFlex DRx System and the subsequent nusinersen 28 mg maintenance dose via the system in the PIERRE study.
Drug: Nusinersen
Administered as specified in the treatment arm.
Other Names:
  • BIIB058
Device: ThecaFlex DRx System
Implanted as specified in the treatment arm.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Maximum Observed Concentration (Cmax) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System
Time Frame: Pre-dose and at multiple time points post-dose over 4 months
Pre-dose and at multiple time points post-dose over 4 months
Area Under the Plasma Concentration-Time Curve From Zero Time to 24 Hours After Intrathecal Administration (AUC0-24h) of Nusinersen Delivered via Standard LP and via ThecaFlex DRx System
Time Frame: Pre-dose and at multiple time points post-dose over 4 months
Pre-dose and at multiple time points post-dose over 4 months

Collaborators and Investigators

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

Sponsor

Biogen

Collaborators

Alcyone Therapeutics, Inc

Investigators

  • Study Director: Medical Director, Biogen

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)

January 16, 2025

Primary Completion (Estimated)

September 30, 2027

Study Completion (Estimated)

September 30, 2027

Study Registration Dates

First Submitted

August 9, 2024

First Submitted That Met QC Criteria

August 13, 2024

First Posted (Actual)

August 15, 2024

Study Record Updates

Last Update Posted (Actual)

September 3, 2026

Last Update Submitted That Met QC Criteria

September 2, 2026

Last Verified

September 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 295SM101
  • 2024-514239-21-00 (Other Identifier: EU CTIS Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

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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