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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)

RecruitingPhase 3Ages 0 Days - 42 DaysAllBiogen
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In plain words

What the trial is, and whether it could suit you

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 changes lower the amount of SMN protein in their bodies. Without enough of this protein, motor neurons and muscles cannot work properly. A similar gene called SMN2 can help replace some of the lost SMN protein in the body. Salanersen works by helping the SMN2 gene to make more SMN protein. In this study, participants will have either 2 SMN2 copies or 3 SMN2 copies. The higher the copy number, the less severe the participant's SMA is. The main goal of this study is to see if starting salanerse

Sign in to get the eligibility rules as simple questions to take to your doctor.

📋 Eligibility rules (as published)
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.
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What it tests

  • Salanersen
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Where

14 sites
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, United States
Neurology Rare Disease Center
Flower Mound, United States
Childrens Hospital of the Kings Daughter Norfolk
Norfolk, United States
Sydney Children's Hospital_Randwick
Randwick, Australia
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Brazil
Hospital da Crianca de Brasilia
Distrito Federal, Brazil
PSEG Centro de Pesquisa Clinica
Sai Paula, Brazil
Instituto Da Criança HC FMUSP
São Paulo, Brazil

…and 6 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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