jRCT2051230096进行中（未招募）不适用
A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 135
- 主要终点
- -
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Single Arm Study
- 干预模型
- Single Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Open(masking Not Used)
入排标准
- 年龄范围
- 15age old over 至 50age old under（—）
- 性别
- All
入选标准
- •Genetic documentation of 5q SMA homozygous 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 consent.
- •Body weight > 20 kg.
- •Received oral risdiplam per the approved label or per the managed access program as follows:
- •Nusinersen-naive participants must have had prior treatment with risdiplam for >= 6 months before enrollment.
- •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 >=
- •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.
- •NOTE: Other protocol defined Inclusion criteria may apply.
排除标准
- •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.
结局指标
主要结局
-
- Change in total RULM score
次要结局
- Incidence of AEs, including SAEs
- Change in clinical laboratory parameters, ECGs, vital signs from baseline
研究者
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