Status:
NOT_YET_RECRUITING
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)
Lead Sponsor:
Biogen
Conditions:
Muscular Atrophy, Spinal
Eligibility:
All Genders
Up to 42 years
Phase:
PHASE3
Brief Summary
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 b...
Detailed Description
For Part 1 of the study, the primary objective is to evaluate the clinical efficacy of salanersen in participants with genetically diagnosed SMA, and the secondary objective is to evaluate safety and ...
Eligibility Criteria
Inclusion
- Key
- ≤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
- 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.
Key Trial Info
Start Date :
January 30 2026
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 29 2032
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT07221669
Start Date
January 30 2026
End Date
May 29 2032
Last Update
December 22 2025
Active Locations (2)
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1
Neurology Rare Disease Center
Flower Mound, Texas, United States, 75028
2
Childrens Hospital of the Kings Daughter Norfolk
Norfolk, Virginia, United States, 23507