Salanersen given before symptoms to help movement in babies with genetic 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 gives salanersen to presymptomatic infants with genetically diagnosed 5q SMA and either two or three SMN2 copies, starting within the first 42 days of life. Participants must meet electrical and growth criteria and have no clinical signs of SMA at baseline. The study measures clinical motor outcomes, long-term safety, pharmacokinetics, and biomarker changes across two parts including extended follow-up. Outcomes will be compared to natural history benchmarks for presymptomatic SMA to determine whether early treatment changes disease onset or severity.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

-≤42 days of age at first dose of salanersen.

* Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation.
* Two or three copies of the survival motor neuron 2 (SMN2) gene.
* Ulnar compound muscle action potential (CMAP) amplitude ≥2 millivolt (mV) at Screening and Day 1 predose.
* Body weight ≥3rd percentile for age based on World Health Organization (WHO) Child Growth Standards at the time of informed consent.

Key Exclusion Criteria:

* Any clinical signs or symptoms at Screening or Day 1 predose that are, in the opinion of the Investigator, strongly suggestive of SMA.
* Areflexia on neurologic examination at biceps, knee, or ankle at Screening or Day 1 Predose.
* Hypoxemia (oxygen saturation \<96% awake or asleep without any supplemental oxygen or respiratory support, or for altitudes \>1000 meters (m), oxygen saturation of \<92% awake or asleep without any supplemental oxygen or respiratory support).
* Diagnosis of neonatal respiratory distress syndrome necessitating surfactant replacement therapy or invasive ventilatory support.
* Any reason, anatomical or otherwise (including hematology/coagulation laboratory results), that presents increased risk of complication from the LP procedures or safety assessments.
* Any prior treatment with an approved SMA disease-modifying therapy (e.g., nusinersen, onasemnogene abeparvovec-xioi \[OA\], and/or risdiplam), a myostatin inhibitor therapy, or an investigational drug given for the treatment of SMA.

Note: Other protocol-defined inclusion/exclusion criteria will apply.

Where it's running

• Ann and Robert H Lurie Childrens Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• Neurology Rare Disease Center
  Flower Mound, Texas, United States Recruiting
• Childrens Hospital of the Kings Daughter Norfolk
  Norfolk, Virginia, United States Recruiting
• Sydney Children's Hospital_Randwick
  Randwick, New South Wales, Australia Recruiting
• Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
  Porto Alegre, Rio Grande do Sul, Brazil Recruiting
• Hospital da Crianca de Brasilia
  Distrito Federal, Brazil 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.