Status:
COMPLETED
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)
Lead Sponsor:
Biogen
Conditions:
Muscular Atrophy, Spinal
Eligibility:
All Genders
2-36 years
Phase:
PHASE4
Brief Summary
In this study, researchers will learn more about the use of nusinersen (BIIB058) in participants with spinal muscular atrophy (SMA). This study will focus on children under the age of 3 who were previ...
Detailed Description
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 abe...
Eligibility Criteria
Inclusion
- Key
- 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
- Key
Exclusion
- 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.
- Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Key Trial Info
Start Date :
January 4 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 9 2025
Estimated Enrollment :
46 Patients enrolled
Trial Details
Trial ID
NCT04488133
Start Date
January 4 2021
End Date
October 9 2025
Last Update
October 14 2025
Active Locations (15)
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1
Arkansas Children's Hospital Research Institute
Little Rock, Arkansas, United States, 72202
2
Stanford Neuromuscular Research
Palo Alto, California, United States, 94304
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
4
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611