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)
Recruiting
Phase 1
Interventional Study
Muscular Atrophy, Spinal
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 3 and older
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 58 (estimated)
- Sponsor
- Biogen · Industry
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About This Trial
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 u…
Trial Locations
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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 …
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