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NCT06555419 · ClinicalTrials.gov registry record · Phase 1
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)
A Phase 1 study of Muscular Atrophy, Spinal, sponsored by Biogen.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 58
- Enrollment target
- 19
- Study locations
NCT06555419 is a Phase 1 study of Muscular Atrophy, Spinal that is actively recruiting participants, run by Biogen. The registered enrollment target is 58 participants, below the 72-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (19% lower). The trial reports 19 study locations across 10 states.
The verdict
NCT06555419, a Phase 1 study of Muscular Atrophy, Spinal, is actively recruiting participants, sponsored by Biogen.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 58 participants
- Enrollment target
- 19
- Study locations
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 |
Interested in This Trial?
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, categorized as Phase 1. The registered enrollment target is 58 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 72-participant average among 15 other Muscular Atrophy, Spinal trials with a reported enrollment target (19% lower). The listed sponsor is Biogen, which has 334 total studies on file at ClinicalTrials.gov.
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.
NCT06555419 reports 19 study locations spanning 10 distinct geographic areas - top geographies include Other, California, Pennsylvania.
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.
What interventions are being tested in trial NCT06555419?
The interventions under investigation include: ThecaFlex DRx System (DEVICE), Nusinersen (DRUG).
Who is sponsoring clinical trial NCT06555419?
This trial is sponsored by Biogen, which has 334 total clinical trials registered on ClinicalTrials.gov.
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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