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)

Launched by BIOGEN · Aug 13, 2024

Keywords

ClinConnect Summary

The PIERRE-PK study is a clinical trial aimed at understanding how the medication nusinersen works in the body when it is delivered using a new device called the ThecaFlex DRx™ System, compared to the traditional method known as a lumbar puncture (LP). This study is specifically for people with spinal muscular atrophy (SMA) who are already receiving nusinersen treatment. To participate, individuals must be on a regular schedule of receiving nusinersen, specifically a 12 mg dose every four months, and must also be enrolled in the larger PIERRE study.

Participants in the PIERRE-PK study can expect to undergo some screening tests to see if they qualify, followed by receiving nusinersen through both the lumbar puncture and the new ThecaFlex device. Researchers will take blood samples before and after each dose to measure how much of the medication is in the bloodstream. The total time commitment for each participant will be up to five months, which overlaps with their involvement in the PIERRE study. It's important to note that individuals who are currently pregnant, breastfeeding, or have recently participated in other SMA studies may not be eligible for this trial.

Gender

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Eligibility criteria

• 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 than 12 mg.
• • Participant has already undergone implantation of the ThecaFlex DRx system.
• • Participant is pregnant, currently breastfeeding, or intending to become pregnant during the study.
• • NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply