Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 2:54 AM
Ignite Modification Date:
2025-12-25 @ 2:54 AM
NCT ID:
NCT04488133
Eligibility Criteria:
Key Inclusion Criteria:
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 Criteria:
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.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
2 Months
Maximum Age:
36 Months
Study:
NCT04488133
Study Brief:
Protocol Section:
NCT04488133