A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)

Recruiting Phase 3 Interventional Study
Muscular Atrophy, Spinal
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
0 Days – 42 Days
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
30 (estimated)
Sponsor
Biogen · Industry
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About This Trial
In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen. Specifically, researchers will learn more about how salanersen works in babies who have been diagnosed with SMA through genetic testing but have not yet started showing signs or symptoms. Most people with SMA have changes in a gene called survival motor neuron 1, also known as SMN1. These…
Trial Locations
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Eligibility Criteria
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) amplitud…
Contacts

US Biogen Clinical Trial Center

866-633-4636

clinicaltrials@biogen.com

CONTACT

Global Biogen Clinical Trial Center

clinicaltrials@biogen.com

CONTACT