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:
2026-03-26 @ 3:15 PM
Ignite Modification Date:
2026-03-26 @ 3:15 PM
NCT ID:
NCT07444450
Eligibility Criteria:
Key Inclusion Criteria:
* Genetic documentation of 5q spinal muscular atrophy (SMA) homozygous gene deletion or mutation or compound heterozygous mutation.
* 2 copies of the survival motor neuron 2 (SMN2) gene.
* Onasemnogene Abeparvovec (OA) dose given at ≤ 42 days of age and screening initiated less than 6 months from OA dosing.
* OA dose given while participant was presymptomatic, per Investigator attestation. For this study, presymptomatic is defined as follows:
* No clinical signs or symptoms at the time of OA dosing that are, in the opinion of the Investigator, strongly suggestive of SMA.
* No absence of tendon reflexes (i.e., absence of all of biceps, knee and ankle tendons) at the time of OA dosing (e.g., Hammersmith Infant Neurological Examination (HINE) Section 1 or equivalent).
* If Compound Muscle Action Potential (CMAP) data is available at the time of dosing, ulnar CMAP amplitude ≥ 2 millivolt (mV).
Key Exclusion Criteria:
* Any unresolved post-OA laboratory abnormalities defined as follows:
* Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) must be less than 2 × Upper Limit of Normal (ULN) while not receiving corticosteroids within 30 days prior to dosing with salanersen or sham procedure (repeat testing may be performed if necessary).
* Evidence of thrombocytopenia, indicated by the platelet count being lower than the normal range for the laboratory.
* Evidence of elevated troponin-I levels, identified as elevated post-OA, and has not returned to the normal range.
* Confirmed demonstration of corrected QT interval, using Fridericia's correction method, of \> 450 milliseconds (ms).
* Other than OA, any prior treatment with an approved SMA disease modifying therapy (e.g. nusinersen and/or risdiplam), a myostatin inhibitor therapy, or an investigational drug given for the treatment of SMA.
* Steroid treatment administered for the purpose of treating complications following OA within 14 days prior to dosing with salanersen or sham procedure on Day 1.
Note: Other protocol-defined inclusion/exclusion criteria will apply.
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
0 Days
Maximum Age:
7 Months
Study:
NCT07444450
Study Brief:
Protocol Section:
NCT07444450