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)
An Open Label Study to Assess the Pharmacokinetic Profile of Nusinersen (BIIB058) Administered Via the ThecaFlex DRx™ System (PIERRE-PK)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Children's Hospital of Orange County — Orange, California
Age range: 3 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (19 Sites)
- Children's Hospital of Orange County — Orange, California
- Stanford University Medical Center | Department of Neurology_Palo Alto — Palo Alto, California
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- Boston Children's Hospital — Boston, Massachusetts
- Helen DeVos Children's Hospita — Grand Rapids, Michigan
- Milton S. Hershey Medical Center | Pennsylvania State University_Hershey — Hershey, Pennsylvania
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Texas Childrens Hospital Houston — Houston, Texas
- Stacey Hall Developmental Pediatrics — Charlottesville, Virginia
- Children's Hospital of the King's Daughters_Norfolk — Norfolk, Virginia
- Hôpital Raymond Poincaré — Garches, Hauts De Seine
- 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,
- Royal Hallamshire Hospital Neurology Department — Sheffield, South Yorkshire
📞 Contact the Study Coordinator
Phone: (866) 633-4636
Email: clinicaltrials@biogen.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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- A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy — Little Rock, Arkansas
- A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec — Flower Mound, Texas
- A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies — Palo Alto, California
Explore by condition: All Muscular Atrophy, Spinal clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.