Phase
Condition
Muscular Dystrophy
Spinal Muscular Atrophy
Myasthenia Gravis (Chronic Weakness)
Treatment
ThecaFlex DRx System
Nusinersen
Clinical Study ID
Ages > 3 All Genders
Study Summary
Eligibility Criteria
Inclusion
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 andthe last nusinersen dose prior to study enrollment.
Participants must be enrolled in the PIERRE study to be eligible for enrolment inthe PIERRE PK study
Exclusion
Key Exclusion Criteria:
Ongoing participation or participation within 6 months or 5 half-lives of the agent (whichever is longer) of enrolment in other interventional clinical trials for thetreatment of SMA (except for the PIERRE study).
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 pregnantduring the study.
NOTE: Other protocol-defined Inclusion/Exclusion criteria may apply
Study Design
Connect with a study center
Texas Childrens Hospital Houston
Houston, Texas 77030
United StatesActive - Recruiting
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