Status:
RECRUITING
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)
Lead Sponsor:
Biogen
Collaborating Sponsors:
Alcyone Therapeutics, Inc
Conditions:
Muscular Atrophy, Spinal
Eligibility:
All Genders
3+ years
Phase:
PHASE1
Brief 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 ...
Detailed Description
The primary objective of this study is to assess the Pharmacokinetic (PK) profile of nusinersen delivered via standard LP and via the ThecaFlex DRx System in participants with SMA.
Eligibility Criteria
Inclusion
- Key
- 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
- 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
Key Trial Info
Start Date :
January 16 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 15 2026
Estimated Enrollment :
58 Patients enrolled
Trial Details
Trial ID
NCT06555419
Start Date
January 16 2025
End Date
May 15 2026
Last Update
September 8 2025
Active Locations (18)
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1
Children's Hospital of Orange County
Orange, California, United States, 92868
2
Stanford University Medical Center | Department of Neurology_Palo Alto
Palo Alto, California, United States, 94304
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611-2605
4
Boston Children's Hospital
Boston, Massachusetts, United States, 02115