Salanersen after early gene therapy in babies with SMA

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 3 interventional trial will evaluate the safety and tolerability of introducing salanersen six months after OA treatment in genetically diagnosed SMA babies who were presymptomatically treated. Secondary goals include measuring efficacy, biomarker changes, and pharmacokinetics of salanersen in this population. Participants must have two SMN2 copies, and OA treatment initiated before six months of age under presymptomatic conditions. The study uses a sham procedure as a control. Lead sponsor is Biogen, with study site at a neurology rare disease center in Flower Mound, Texas.

Why it matters

Who can join: the full rules

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.

Where it's running

• Neurology Rare Disease Center
  Flower Mound, Texas, United States Recruiting

Study contacts

• Study coordinator: US Biogen Clinical Trial Center
• Email: clinicaltrials@biogen.com
• Phone: 866-633-4636

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.