Actively Recruiting

Phase 1
Age: 3Years +
All Genders
NCT06555419

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)

Led by Biogen · Updated on 2026-03-18

58

Participants Needed

19

Research Sites

127 weeks

Total Duration

On this page

Sponsors

B

Biogen

Lead Sponsor

A

Alcyone Therapeutics, Inc

Collaborating Sponsor

AI-Summary

What this Trial 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 objective 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 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. * 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

Official Title

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)

Who Can Participate

Age: 3Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participant is on regular maintenance dosing of nusinersen (12 milligrams [mg] dose) every 4 months, with 4 months (± 2 weeks) between the LP-delivered study dose and the last nusinersen dose prior to study enrollment.
  • Participants must be enrolled in the PIERRE study to be eligible for enrolment in the PIERRE PK study.
  • Participant is 3 years old or older.
Not Eligible

You will not qualify if you...

  • Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) in other interventional clinical trials for SMA treatment (except the PIERRE study or myostatin inhibitor trials).
  • Participant is naïve to nusinersen treatment.
  • Participant is receiving nusinersen at a dose other than 12 mg.
  • Participant has already undergone implantation of the ThecaFlex DRx system.
  • Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 19 locations

1

Children's Hospital of Orange County

Orange, California, United States, 92868

Actively Recruiting

2

Stanford University Medical Center | Department of Neurology_Palo Alto

Palo Alto, California, United States, 94304

Actively Recruiting

3

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

Chicago, Illinois, United States, 60611-2605

Actively Recruiting

4

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

Actively Recruiting

5

Helen DeVos Children's Hospita

Grand Rapids, Michigan, United States, 49503

Actively Recruiting

6

Milton S. Hershey Medical Center | Pennsylvania State University_Hershey

Hershey, Pennsylvania, United States, 17033

Actively Recruiting

7

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

8

Texas Childrens Hospital Houston

Houston, Texas, United States, 77030

Actively Recruiting

9

Stacey Hall Developmental Pediatrics

Charlottesville, Virginia, United States, 22903

Actively Recruiting

10

Children's Hospital of the King's Daughters_Norfolk

Norfolk, Virginia, United States, 23507

Actively Recruiting

11

Hôpital Raymond Poincaré

Garches, Hauts De Seine, France, 92380

Actively Recruiting

12

Universitaetsklinikum Essen

Essen, Germany, 45147

Actively Recruiting

13

Fondazione Serena Onlus - Centro Clinico Nemo_Milano

Milan, Italy, 20162

Withdrawn

14

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Italy, 168

Withdrawn

15

Szpital Specjalistyczny im. L.Rydygiera w Krakowie

Krakow, Poland, 31-826

Not Yet Recruiting

16

Instytut Centrum Zdrowia Matki Polki

Lodz, Poland, 93-338

Not Yet Recruiting

17

Hospital Universitario La Paz

Madrid, Spain, 28046

Actively Recruiting

18

Hospital Universitari i Politecnic La Fe_Valencia

Valencia, Spain, 46026

Actively Recruiting

19

Royal Hallamshire Hospital Neurology Department

Sheffield, South Yorkshire, United Kingdom, S102JF

Actively Recruiting

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Research Team

U

US Biogen Clinical Trial Center

CONTACT

G

Global Biogen Clinical Trial Center

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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