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A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
Recruiting
I'm InterestedTrial ID: NCT04488133
Purpose
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; and
clinical outcomes following treatment with Nusinersen in participants with SMA who previously
received onasemnogene abeparvovec.
Official Title
A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec
Stanford Investigator(s)
John W. Day, MD, PhD
Professor of Neurology, of Pediatrics (Genetics) and, by courtesy, of Pathology
Eligibility
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:
- ≤9 months of age (270 days) 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.
Intervention(s):
drug: Nusinersen
Recruiting
I'm InterestedContact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
650-725-4341