A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
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Keep this study
Lead
Biogen
The primary objective of this study is to evaluate the clinical outcomes following treatment with nusinersen in participants with spinal muscular atrophy (SMA) who previously received onasemnogene abeparvovec.
The secondary objectives of this study are to evaluate the safety and tolerability; clinical outcomes and pharmacodynamics (PD) of nusinersen treatment in participants with SMA who previously received onasemnogene abeparvovec.
Age
0–3
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
For all participants:
Genetic documentation of 5q SMA homozygous gene survival motor neuron 1 (SMN1) deletion or mutation, or compound heterozygous mutation
SMN2 copy number of ≥1
≤36 months of age at the time of first Nusinersen dose
Must have previously received onasemnogene abeparvovec per the approved label or local/regional regulations ≥2 months prior to first Nusinersen dose
Must have suboptimal clinical status per the Investigator
Additional Criteria for Subgroups A and B:
\<300 days of age at the time of first Nusinersen dose
SMN2 copy number of 2
Additional Criteria for Subgroup A:
SMA symptom onset ≤4 months (120 days) of age
Must have received intravenous (IV) onasemnogene abeparvovec at \>6 weeks to ≤6 months (43 days to 180 days) of age
Must have received IV onasemnogene abeparvovec after SMA symptom onset
Additional Criteria for Subgroup B:
Must have received IV onasemnogene abeparvovec at ≤6 weeks (42 days) of age
You may not be if
For all participants:
Prior exposure to Nusinersen
Ongoing severe or serious AEs related to onasemnogene abeparvovec
Treatment with an investigational drug, biological agent, or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to study; any prior or current treatment with any survival motor neuron 2 (SMN2)-directed splicing modifier; prior antisense oligonucleotide treatment or cell transplantation; gene therapy for the treatment of SMA other than onasemnogene abeparvovec. Note: treatment with onasemnogene abeparvovec as part of an investigational study is allowed
Additional Criteria for Subgroups A and B:
Weight-for-age is below the third percentile, based on WHO Child Growth Standards at the time of receiving onasemnogene abeparvovec. Adjustments for the gestational weight of premature babies enrolled in Subgroups A and B are allowed provided IV onasemnogene abeparvovec was dosed per the approved label or per local/regional regulations.
Clareo Health | A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
