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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)

RecruitingPhase 1Ages 3 Years - anyAllBiogen
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In plain words

What the trial is, and whether it could suit you

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?

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
Key Inclusion Criteria: \- Participants must be screened in the PIERRE study to be eligible for enrollment in the PIERRE PK study. Participants in the 12 mg group * Participant must have completed all 4 required 12 mg loading doses as per the approved 12 mg dose regimen. * Participant is on regular LP maintenance dosing of nusinersen 12 mg every 4 months, with 120 days ± 14 days between the LP-delivered study dose and the last nusinersen dose prior to study enrollment. Participants in the 28 mg group * Participant must have completed 1 or 2 of the 50 mg loading doses as per the approved 50/28 mg dose regimen. * Participant is on regular LP maintenance dosing of nusinersen 28 mg every 4 months, with 120 days ± 14 days between the LP-delivered study dose and the last nusinersen dose prior to study enrollment. 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 or 28 mg. * Participant has already undergone implantation of the ThecaFlex DRx system or any other implantable medical device for intrathecal (IT) administration. * Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study. NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply.
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What it tests

  • Nusinersen
  • ThecaFlex DRx System
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Where

21 sites
St. Joseph's Hospital and Medical Center - Barrow Neurological Institute
Phoenix, United States
Children's Hospital of Orange County
Orange, United States
Stanford University Medical Center | Department of Neurology_Palo Alto
Palo Alto, United States
Nemours Children's Hospital, Orlando
Orlando, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
Boston Children's Hospital
Boston, United States
Helen DeVos Children's Hospita
Grand Rapids, United States
Milton S. Hershey Medical Center | Pennsylvania State University_Hershey
Hershey, United States

…and 13 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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