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.
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