NCT06555419 · Biogen
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)
(PIERRE-PK)
What this 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 experimental implantable medical device developed by Alcyone Therapeutics, Inc.
View original scientific description
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 can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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
Where
- Orange, California
- Palo Alto, California
- Chicago, Illinois
- Boston, Massachusetts
- Grand Rapids, Michigan
- Hershey, Pennsylvania
- Philadelphia, Pennsylvania
- Houston, Texas
- Charlottesville, Virginia
- Norfolk, Virginia
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 18, 2026 · Source of record for eligibility and locations