Recruiting
Phase 1

Nusinersen

Sponsor:

Biogen

Code:

NCT06555419

Conditions

Muscular Atrophy, Spinal

Eligibility Criteria

Sex: All

Age: 3+

Healthy Volunteers: Not accepted

Interventions

Nusinersen

ThecaFlex DRx System

Study Details

Brief summary:

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.

Conditions

Muscular Atrophy, Spinal

Study ID

NCT06555419

Start date

Jan 16, 2025

Status verified date

Sep, 2026

Completion date

Sep 30, 2027

Anticipated

Primary completion date

Sep 30, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 3+

Healthy Volunteers: Not accepted

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 Design

Enrollment

78 participants

Anticipated

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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.

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.

Interventions

Nusinersen

Administered as specified in the treatment arm.

ThecaFlex DRx System

Implanted as specified in the treatment arm.

Primary outcome measure

  • 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 ]
  • 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 ]

Central Contacts and Locations

Central contacts

US Biogen Clinical Trial Center

866-633-4636clinicaltrials@biogen.com

Global Biogen Clinical Trial Center

clinicaltrials@biogen.com

Locations

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

Recruiting

Phoenix, Arizona, United States, 85013

Contacts

602-406-6262

Principal Investigator:

Shafeeq Ladha

Children's Hospital of Orange County

Recruiting

Orange, California, United States, 92868

Contacts

714-509-3605

Principal Investigator:

Julian Thomas

Stanford University Medical Center | Department of Neurology_Palo Alto

Recruiting

Palo Alto, California, United States, 94304

Contacts

650-723-5152

Principal Investigator:

John Day

Nemours Children's Hospital, Orlando

Recruiting

Orlando, Florida, United States, 32827

Contacts

407-650-7715

Principal Investigator:

Abdul Hamid Omer

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

Recruiting

Chicago, Illinois, United States, 60611-2605

Contacts

312-227-3550

Principal Investigator:

Nancy Kuntz

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

617-355-8036

Principal Investigator:

Basil T Darras

Helen DeVos Children's Hospita

Recruiting

Grand Rapids, Michigan, United States, 49503

Contacts

616-391-2241

Principal Investigator:

Jena Krueger

Milton S. Hershey Medical Center | Pennsylvania State University_Hershey

Recruiting

Hershey, Pennsylvania, United States, 17033

Contacts

717-232-5443

Principal Investigator:

Dustin Paul

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

215-590-3174

Principal Investigator:

Patrick Cahill

Texas Childrens Hospital Houston

Recruiting

Houston, Texas, United States, 77030

Contacts

832-822-1750

Principal Investigator:

David Bauer

Stacey Hall Developmental Pediatrics

Recruiting

Charlottesville, Virginia, United States, 22903

Contacts

434-924-8184

Principal Investigator:

Rebecca J Scharf

Children's Hospital of the King's Daughters_Norfolk

Recruiting

Norfolk, Virginia, United States, 23507

Contacts

757-668-7000

Principal Investigator:

Crystal Proud

More Information

Sponsor

Biogen

Last update posted

Sep 3, 2026

Last verified

Sep, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Biogen on 2026-09-03.