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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)

Biogen · with Alcyone Therapeutics, Inc · NCT06555419

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 3+ · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1
Target enrollment
78
US sites recruiting
12
Study start
January 16, 2025
Record last updated
September 3, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: Nusinersen; ThecaFlex DRx System

Conditions: Muscular Atrophy, Spinal

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

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.

Recruiting US locations

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

    Phoenix, AZ 85013

    Site contact: 602-406-6262

  • Children's Hospital of Orange County

    Orange, CA 92868

    Site contact: 714-509-3605

  • Stanford University Medical Center | Department of Neurology_Palo Alto

    Palo Alto, CA 94304

    Site contact: 650-723-5152

  • Nemours Children's Hospital, Orlando

    Orlando, FL 32827

    Site contact: 407-650-7715

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

    Chicago, IL 60611-2605

    Site contact: 312-227-3550

  • Boston Children's Hospital

    Boston, MA 02115

    Site contact: 617-355-8036

  • Helen DeVos Children's Hospita

    Grand Rapids, MI 49503

    Site contact: 616-391-2241

  • Milton S. Hershey Medical Center | Pennsylvania State University_Hershey

    Hershey, PA 17033

    Site contact: 717-232-5443

  • Children's Hospital of Philadelphia

    Philadelphia, PA 19104

    Site contact: 215-590-3174

  • Texas Childrens Hospital Houston

    Houston, TX 77030

    Site contact: 832-822-1750

  • Stacey Hall Developmental Pediatrics

    Charlottesville, VA 22903

    Site contact: 434-924-8184

  • Children's Hospital of the King's Daughters_Norfolk

    Norfolk, VA 23507

    Site contact: 757-668-7000

Study contacts (from the official record)

  • US Biogen Clinical Trial Center · 866-633-4636 · clinicaltrials@biogen.com
  • Global Biogen Clinical Trial Center · clinicaltrials@biogen.com
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A Study to Find Out How Nusinersen is Processed in the Body When… (NCT06555419) | Bored Studies