Last updated 5 days ago
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)
30 patients around the world
Available in United States, Brazil
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 tolerability, pharmacokinetics (PK), and effect on biomarkers after treatment with salanersen in participants with genetically diagnosed SMA.
For Part 2 of the study, the primary objective is to evaluate the long-term clinical efficacy of salanersen in participants with genetically diagnosed SMA, and the secondary objective is to evaluate the long-term safety and tolerability, PK, and effect on biomarkers after treatment with salanersen in participants with genetically diagnosed SMA.
Biogen
1Research sites
30Patients around the world
This study is for people with
Rare diseases
Spinal muscular atrophy
Requirements for the patient
To 42 Days
All Gender
Medical requirements
- ≤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.
- 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.
Sites
Hospital da Criança de Brasília José Alencar
Recruiting
StudySTELLAR-1
SponsorBiogen
Study typeInterventional
Conditions
Spinal muscular atrophy
Requirements
To 42 DaysAll Gender
Unique study IDclinicaltrials.gov
NCT07221669
