RecruitingPhase 1NCT06555419

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)

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


Sponsor

Biogen

Enrollment

78 participants

Start Date

Jan 16, 2025

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 3 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Nusinersen and a medical device called ThecaFlex DRx System for people with muscular atrophy, spinal. The study is currently recruiting participants at 19 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGNusinersen

Administered as specified in the treatment arm.

DEVICEThecaFlex DRx System

Implanted as specified in the treatment arm.


Locations(21)

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

Phoenix, Arizona, United States

Children's Hospital of Orange County

Orange, California, United States

Stanford University Medical Center | Department of Neurology_Palo Alto

Palo Alto, California, United States

Nemours Children's Hospital, Orlando

Orlando, Florida, United States

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

Chicago, Illinois, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Helen DeVos Children's Hospita

Grand Rapids, Michigan, United States

Milton S. Hershey Medical Center | Pennsylvania State University_Hershey

Hershey, Pennsylvania, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Texas Childrens Hospital Houston

Houston, Texas, United States

Stacey Hall Developmental Pediatrics

Charlottesville, Virginia, United States

Children's Hospital of the King's Daughters_Norfolk

Norfolk, Virginia, United States

Hôpital Raymond Poincaré

Garches, Hauts De Seine, France

Universitaetsklinikum Essen

Essen, Germany

Fondazione Serena Onlus - Centro Clinico Nemo_Milano

Milan, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Italy

Szpital Specjalistyczny im. L.Rydygiera w Krakowie

Krakow, Poland

Instytut Centrum Zdrowia Matki Polki

Lodz, Poland

Hospital Universitario La Paz

Madrid, Spain

Hospital Universitari i Politecnic La Fe_Valencia

Valencia, Spain

Royal Hallamshire Hospital Neurology Department

Sheffield, South Yorkshire, United Kingdom

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NCT06555419


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