Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

BIOGEN IDEC RESEARCH LIMITED0 个研究点目标入组 152 人开始时间: 2021年6月17日最近更新: 2024年6月24日

研究概览

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•Part A, B and C: - Genetic documentation of 5q SMA (homozygous gene deletion, mutation, or compound heterozygote)
•- Participants with SMA symptom onset = 6 months (= 180 days) of age (infantile onset) should have age = 7 months (= 210 days) at the time of informed consent
•- Participants with SMA symptom onset > 6 months (> 180 days) of age (later onset):
•- Age 2 to < 10 years at the time of informed consent
•- Can sit independently but has never had the ability to walk independently
•- HFMSE score = 10 and = 54 at Screening
•- Participants = 18 years of age at Screening must be ambulatory
•- Currently on nusinersen treatment at the time of Screening, with the first dose being at least 1 year prior to Screening
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 12
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•Part A, B and C:
•- 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 (CNS) catheter
•- Hospitalization for surgery, pulmonary event, or nutritional support within 2 months prior to Screening or planned within 12 months after the participant’s first dose
•- Dosing with onasemnogene abeparvovec-xioi (Zolgensma) within 6 months prior to Screening
•- Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for > 6 hours during a 24-hour period, at Screening
•- Medical necessity for a gastric feeding tube
•- Treatment with an investigational drug given for the treatment of SMA, biological agent, or device within 30 days prior to Screening or anytime during the study; prior treatment with risdiplam or branaplam; or any history of gene therapy, prior antisense oligonucleotide treatment, or cell transplantation
•- Concurrent participation and/or administration of nusinersen in another clinical study
•NOTE: Other protocol defined Inclusion/Exclusion criteria may apply

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