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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)
NCT06555419 · View on ClinicalTrials.gov ↗
Study 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 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 Studied
Interventions
- DEVICE ThecaFlex DRx System
- DRUG Nusinersen
Study Locations (19)
Other
- Universitaetsklinikum Essen — Essen
- Fondazione Serena Onlus - Centro Clinico Nemo_Milano — Milan
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Roma
- Szpital Specjalistyczny im. L.Rydygiera w Krakowie — Krakow
- Instytut Centrum Zdrowia Matki Polki — Lodz
- Hospital Universitario La Paz — Madrid
- Hospital Universitari i Politecnic La Fe_Valencia — Valencia
California
- Children's Hospital of Orange County — Orange
- Stanford University Medical Center | Department of Neurology_Palo Alto — Palo Alto
Pennsylvania
- Milton S. Hershey Medical Center | Pennsylvania State University_Hershey — Hershey
- Children's Hospital of Philadelphia — Philadelphia
Virginia
- Stacey Hall Developmental Pediatrics — Charlottesville
- Children's Hospital of the King's Daughters_Norfolk — Norfolk
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
Massachusetts
- Boston Children's Hospital — Boston
Michigan
- Helen DeVos Children's Hospita — Grand Rapids
Texas
- Texas Childrens Hospital Houston — Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 58 participants |
| Start Date | 2025-01-16 |
| Est. Completion | 2027-06-25 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06555419
The ClinicalTrials.gov registry entry for NCT06555419 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 58 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Biogen, which has 74 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Muscular Atrophy, Spinal appearing as the primary indexed condition, and to 2 interventions — of which ThecaFlex DRx System is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06555419 reports 19 study locations spanning 10 distinct geographic areas — top geographies include Other, California, Pennsylvania. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06555419 about?
NCT06555419 is a clinical study titled "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)". 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 Therapeut...
What is the current status of trial NCT06555419?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 58 participants. The study started on 2025-01-16. Estimated completion is 2027-06-25.
What conditions does trial NCT06555419 study?
This clinical trial studies the following conditions: Muscular Atrophy, Spinal. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06555419?
The interventions under investigation include: ThecaFlex DRx System (DEVICE), Nusinersen (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06555419?
This trial is sponsored by Biogen, which has 74 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06555419 being conducted?
This trial has 19 study locations across California, Illinois, Massachusetts, Michigan, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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