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About
The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy (SMA) previously treated with risdiplam.
The secondary objective of this study is to evaluate the safety and tolerability of HD nusinersen in participants with SMA previously treated with risdiplam.
Enrollment
Sex
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Volunteers
Inclusion and exclusion criteria
Key Inclusion Criteria:
Genetic documentation of 5q SMA homozygous survival motor neuron-1 (SMN1) gene deletion or mutation or compound heterozygous mutation.
Diagnosis of later-onset SMA with symptom onset at age >6 months.
Aged ≥15 to ≤50 years at the time of informed consent
Body weight >20 kg.
Received oral risdiplam per the approved label or per the managed access program as follows
Able to perform the age-appropriate functional assessments in the study.
RULM entry item A score ≥3.
RULM total score ≥5 and ≤30 at Screening.
Nonambulatory, defined as not able to walk 15 feet (4.57 meters) independently without support.
Willing to stop risdiplam treatment.
Willing and able to start treatment with HD nusinersen.
Key Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Primary purpose
Allocation
Interventional model
Masking
45 participants in 1 patient group
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Central trial contact
US Biogen Clinical Trial Center; Global Biogen Clinical Trial Center
Data sourced from clinicaltrials.gov
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