Nusinersen dosing study via flexible spinal catheter
Part of Brain & nervous system clinical trials.
This trial investigates how the drug nusinersen (Spinraza) enters the body when given through a small, flexible tube placed in the lower back (ThecaFlex DRx system). It aims to learn how the drug is processed in adults and children with spinal muscular atrophy, which could help make dosing more convenient.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be already enrolled in the PIERRE study.
- If you take the 12 mg dose, you must have completed all 4 loading doses and have been on regular maintenance dosing every 4 months.
- If you take the 28 mg dose, you must have completed 1 or 2 of the 50 mg loading doses and be on regular maintenance dosing every 4 months.
- You must not have used other experimental treatments for SMA in the last 6 months, except for myostatin inhibitors or the PIERRE study.
- You must not have previously had a ThecaFlex or similar implant.
- You must not be pregnant, breastfeeding, or planning pregnancy during the study.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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