CTIS2023-505643-39-00招募中1 期
A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending 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. - 277HV101
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 62
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 64（—）
- 性别
- All
入选标准
- •Part A - Male healthy participants aged 18 to 55 years, inclusive., Part B - Potential for improvement due to suboptimal clinical status secondary to SMA, as determined by the Investigator., Other protocol defined Inclusion criteria may apply., Part A - Have a body mass index of 18 to 30 kilograms per meter square (kg/m^2), inclusive., Part A - Must be in good health as determined by the investigator, based on medical history and screening evaluations., Part B - Age 0.5 to 12 years old, inclusive, at the time of informed consent., Part B - Weight =7 kg at the time of informed consent., Part B - Genetic diagnosis of SMA (5q SMA homozygous survival motor neuron 1 (SMN1) gene deletion or mutation or compound heterozygous mutation)., Part B - Survival motor neuron 2 (SMN2) copy number =1., Part B - Must have received intravenous (IV) onasemnogene abeparvovec per the approved label or per guidelines including the steroid regimen and monitoring specified therein., Part B - Treatment with onasemnogene abeparvovec =180 days prior to first BIIB115 dose.
排除标准
- •Part A - Any reason, anatomical or otherwise (including abnormal hematology/coagulation), that presents increase of risk of complication from multiple lumbar puncture (LP) procedures required for dosing and CSF collection, per the investigator discretion., Part B - Treatment with any SMN2-splicing modifier (nusinersen or risdiplam) after receiving onasemnogene abeparvovec. Treatment with nusinersen <12 months from the first dose of BIIB115., Part B - 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., Part B - Permanent ventilation, defined as tracheostomy or =16 hours ventilation /day continuously for >21 days in the absence of an acute reversible event., Other protocol defined Exclusion criteria may apply., Part A - History of any clinically significant cardiac, endocrine, gastrointestinal, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, or renal disease, or other major disease, as determined by the Investigator., Part A - Chronic, recurrent, or serious infection, as determined by the investigator, within 90 days prior to screening or between screening and Day -1., Part A - Current enrollment or a plan to enroll in any interventional clinical study of a drug, biologic, or device, in which an investigational treatment or approved therapy for investigational use is administered within 3 months (or 5 half-lives of the agent, whichever is longer) prior to randomization., Part B - Severe or serious AEs related to onasemnogene abeparvovec therapy that are ongoing during Screening., Part B - Interval of <180 days between onasemnogene abeparvovec therapy and first BIIB115 dose., Part B - Ongoing steroid treatment following onasemnogene abeparvovec at time of screening., Part B - History of drug induced liver injury or liver failure per Hy’s law definition., Part B - History of thrombotic micrangiopathy.
研究者
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尚未招募
不适用
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