Last updated 10 months ago
A Study to Evaluate Higher Dose (HD) Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Previously Treated With Risdiplam
45 patients around the world
Available in United States, Brazil, Spain
Biogen
2Research sites
45Patients around the world
Requirements for the patient
To 50 Years
All Gender
Medical requirements
- Genetic documentation of 5q SMA homozygous survival motor neuron-1 (SMN1) gene deletion or mutation or compound heterozygous mutation.Diagnosis of later-onset SMA with symptom onset at age >6 months.Aged ≥15 to ≤50 years at the time of informed consentBody weight >20 kg.Received oral risdiplam per the approved label or per the managed access program as follows 1. Nusinersen-naive participants must have had prior treatment with risdiplam for ≥6 months before enrollment. 2. Nusinersen-experienced participants must have stopped nusinersen for ≥16 months and must have been on risdiplam for ≥12 months before enrollment.Able to perform the age-appropriate functional assessments in the study.RULM entry item A score ≥3.RULM total score ≥5 and ≤30 at Screening.Nonambulatory, defined as not able to walk 15 feet (4.57 meters) independently without support.Willing to stop risdiplam treatment.Willing and able to start treatment with HD nusinersen.
- Any major illness within 1 month before the screening examination or within 1 week prior to Screening and up to first dose administration.Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy at any time during the Screening Period.Presence of an implanted shunt for the drainage of CSF or of an implanted central nervous system catheter.Permanent tracheostomy or permanent ventilation at Screening.The medical necessity, as defined by the Investigator, for noninvasive ventilation such as bilevel positive airway pressure or continuous positive airway pressure outside of regular sleep hours for any reason other than proactive SMA management, at Screening.History of bacterial meningitis, viral encephalitis, or hydrocephalus.Ongoing medical condition that according to the Investigator would interfere with the conduct and assessments of the study. An example is a medical disability (e.g., wasting or cachexia, severe anemia, and respiratory parameters) that would interfere with the assessment of safety or would compromise the ability of the participant to undergo study procedures.Participants who are pregnant or currently breastfeeding and those intending to become pregnant during the study.Treatment with an investigational drug, biological agent, or device within 30 days or 5 halflives of the agent, whichever is longer, prior to Screening or anytime during the study; any prior or current treatment with gene therapy for the treatment of SMA.
Sites
Hospital de Clínicas de Porto Alegre - HCPA/UFRGS
Recruiting
Hospital Universitari i Politecnic La Fe
Recruiting
StudyASCEND
SponsorBiogen
Study typeInterventional
Requirements
To 50 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT05067790
