Condition: Muscular Atrophy, Spinal · Sponsor: Biogen
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 tak…
This description comes directly from the study's public registry record.
US Biogen Clinical Trial Center · 866-633-4636 · clinicaltrials@biogen.com
Global Biogen Clinical Trial Center · clinicaltrials@biogen.com
Always discuss trial participation with your own doctor first.
| Children's Hospital of Orange County | Orange, California, United States | Recruiting |
| Stanford University Medical Center | Department of Neurology_Palo Alto | Palo Alto, California, United States | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago, Illinois, United States | Recruiting |
| Boston Children's Hospital | Boston, Massachusetts, United States | Recruiting |
| Helen DeVos Children's Hospita | Grand Rapids, Michigan, United States | Recruiting |
| Milton S. Hershey Medical Center | Pennsylvania State University_Hershey | Hershey, Pennsylvania, United States | Recruiting |
| Children's Hospital of Philadelphia | Philadelphia, Pennsylvania, United States | Recruiting |
| Texas Childrens Hospital Houston | Houston, Texas, United States | Recruiting |
| Stacey Hall Developmental Pediatrics | Charlottesville, Virginia, United States | Recruiting |
| Children's Hospital of the King's Daughters_Norfolk | Norfolk, Virginia, United States | Recruiting |
| Hôpital Raymond Poincaré | Garches, Hauts De Seine, France | Recruiting |
| Universitaetsklinikum Essen | Essen, Germany | Recruiting |
+ 7 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT06555419