A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFl
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 consist
| Condition(s) | Muscular Atrophy, Spinal |
|---|---|
| Status | Recruiting |
| Phase | Phase 1 |
| Study type | Interventional |
| 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 rese |
| Who can participate | Key Inclusion Criteria: * Participant is on regular maintenance dosing of nusinersen (12 milligrams \[mg\] dose) every 4 months, with 4 months (± 2 weeks) between the LP-delivered study dose and the last nusinersen dose prior to study enrollment. * Participants must be enrolled in the PIERRE study to be eligible for enrolment in the PIERRE PK study. 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 th |
| Ages | 3 Years |
| Sex | All |
| Lead sponsor | Biogen |
| Locations | Orange, California, United States; Palo Alto, California, United States; Chicago, Illinois, United States; Boston, Massachusetts, United States; Grand Rapids, Michigan, United States; Hershey, Pennsylvania, United States (+13 more sites) |
| Start date | 2025-01-16 |
| NCT ID | NCT06555419 |
| Official listing | https://clinicaltrials.gov/study/NCT06555419 |