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About
The primary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with spinal muscular atrophy (SMA), as measured by change in Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) total score (Part B); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C).
The secondary objectives of this study are to examine the clinical efficacy of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A, B and C); to examine the effect of nusinersen administered intrathecally at higher doses to participants with SMA (Parts A and C); to examine the safety and tolerability of nusinersen administered intrathecally at higher doses to participants with SMA, to examine the effect of nusinersen administered intrathecally at higher doses compared to the currently approved dose in participants with SMA (Part B).
Enrollment
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Inclusion and exclusion criteria
Key Inclusion Criteria:
Part A, B and C:
Part A:
Part B:
Participants with SMA symptom onset ≤ 6 months (≤ 180 days) of age (infantile onset) should have age > 1 week to ≤ 7 months (≤ 210 days) at the time of informed consent
Participants with SMA symptom onset > 6 months (> 180 days) of age (later onset):
Part C:
Part C Cohort 1:
Part C Cohort 2:
Key Exclusion Criteria:
Part A, B and C:
Part A:
Part B:
Treatment with an investigational drug including but not limited to the treatment of SMA, biological agent, or device within 30 days or 5 half-lives of the agent, whichever is longer, prior to Screening or anytime during the study; any prior or current treatment with any SMN2-splicing modifier or gene therapy; or prior antisense oligonucleotide treatment, or cell transplantation
Participants with SMA symptom onset > 6 months (> 180 days) of age (later onset):
Participants with SMA symptom onset ≤ 6 months (≤ 180 days) of age (infantile onset): Signs or symptoms of SMA present at birth or within the first week after birth
Part C:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Primary purpose
Allocation
Interventional model
Masking
145 participants in 4 patient groups
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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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