A Randomized, Blinded, Placebo-Controlled, Phase 1 SingleAscending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated with Onasemnogene Abeparvovec (Zolgensma™) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115

Biogen Idec Research Limited0 个研究点目标入组 24 人开始时间: 2023年2月2日最近更新: 2024年4月22日

研究概览

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•- Age 0.5 to 12 years old, inclusive, at the time of informed consent
•- Weight =7 kg at the time of informed consent
•- Genetic diagnosis of SMA (5q SMA homozygous survival motor neuron 1 (SMN1)gene deletion or mutation or compound heterozygous mutation)- Survival motor neuron 2 (SMN2) copy number =1
•- Must have received IV onasemnogene abeparvovec per the approved label or per guidelines including the steroid regimen and monitoring specified therein
•- Treatment with onasemnogene abeparvovec =180 days prior to first BIIB115 dose
•- Potential for improvement due to suboptimal clinical status secondary to SMA, as determined by the Investigator
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 24
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•- Severe or serious AEs related to onasemnogene abeparvovec therapy that are ongoing during Screening
•- Interval of <180 days between onasemnogene abeparvovec therapy and first BIIB115 dose
•- Ongoing steroid treatment following onasemnogene abeparvovec at time of screening
•- History of drug induced liver injury or liver failure per Hy's law definition
•- History of thrombotic micrangiopathy
•- Treatment with any SMN2-splicing modifier (nusinersen or risdiplam) after receiving onasemnogene abeparvovec. Treatment with nusinersen <12 months from the first dose of BIIB115.
•- Any reason, anatomical or otherwise (including abnormal hematology/coagulation),that presents increase of risk of complication from the LP procedures, CSF circulation, or safety assessments, including a history of hydrocephalus or implanted shunt for CSF drainage
•- Permanent ventilation, defined as tracheostomy or =16 hours ventilation/day continuously for =21 days in the absence of an acute reversible event
•NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

发起方