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RECRUITINGPhase 1INTERVENTIONAL

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, Single Cohort Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Key Who May Qualify: - 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. Key Who Should NOT Join This Trial: - 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. - Participant has already undergone implantation of the ThecaFlex DRx system. - Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study. NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Key Inclusion Criteria: * 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. 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. * Participant has already undergone implantation of the ThecaFlex DRx system. * Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study. NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply

Treatments Being Tested

DRUG

Nusinersen

Administered as specified in the treatment arm.

DEVICE

ThecaFlex DRx System

Implanted as specified in the treatment arm.

Locations (19)

Children's Hospital of Orange County
Orange, California, United States
Stanford University Medical Center | Department of Neurology_Palo Alto
Palo Alto, California, 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