NCT07846696尚未招募3 期
An Open Label Individual Patient (OLIP) Study of Nusinersen (BIIB058) in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's1 个研究点 分布在 1 个国家目标入组 1 人开始时间: 2026年9月17日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Long-term safety and tolerability of Nusinersen 28mg dosing through adverse events experienced
研究概览
简要总结
An Open Label Individual Patient (OLIP) Study of Nusinersen (BIIB058) in a Participant with Spinal Muscular Atrophy Who Previously Participated in an Investigational Study with Nusinersen
详细描述
This is a Single Patient Study (SPS)/Open Label Individual Patient (OLIP) long-term extension study to evaluate the safety, tolerability, and efficacy of nusinersen administered by intrathecal injection at a higher dose (28 mg maintenance) for up to 5 years to a participant who has previously participated in Biogen Study 232SM302.
研究设计
- 研究类型
- 干预性
- 分配方式
- 不适用
- 干预模型
- 单组
- 主要目的
- 治疗
- 盲法
- 开放（无盲法）
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- Signed informed consent of parent(s) or legal guardian(s) and signed informed assent of the participant, if indicated per participant's capacity to assent and institutional guidelines.
- Completion of Study 232SM302.
- Ability to complete all study procedures, measurements, and visits, and parent(s) or legal guardian(s)/participant has adequately supportive psychosocial circumstances, in the opinion of the Investigator.
- Meets age-appropriate institutional criteria for the use of anesthesia/sedation, if use is planned for study procedures (as assessed by the Investigator and either anesthesiologist or pulmonologist).
- All female participants of childbearing potential (defined as any female physiologically capable of becoming pregnant) and all male participants of reproductive age must practice effective contraception.
排除标准
- Any new condition or worsening of an existing condition that, according to the Investigator, would make the participant unsuitable for inclusion, could interfere with the assessment of safety, or would compromise the ability of the participant to undergo study procedures.
- Treatment with another investigational therapy or enrollment in another interventional clinical study after the final dose in Study 232SM302.
- Treatment with an approved therapy for SMA after the final dose in Study 232SM302.
- History of systemic hypersensitivity reaction to nusinersen, the excipients contained in the formulation, and if appropriate, any diagnostic agents to be administered during the study.
- Participants who are pregnant or currently breastfeeding and those intending to become pregnant during the study.
- The participant's parent(s) or legal guardian(s) is not willing or able to meet guidelines in the consensus statement for standard of care in SMA [Finkel 2018; Mercuri 2018] or provide nutritional and respiratory support throughout the study, per the Investigator's judgment.
- Other unspecified reasons that, in the opinion of the Investigator, make the participant unsuitable for enrollment.
研究组 & 干预措施
Open Label
Experimental
干预措施: Nusinersen Injectable Product (Drug)
结局指标
主要结局
Long-term safety and tolerability of Nusinersen 28mg dosing through adverse events experienced
时间窗: From enrollment to the end of treatment, one year
次要结局
未报告次要终点
研究者
Craig Campbell
Paediatric Neurologist
London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
研究点 (1)
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标识符
- NCT 编号
- NCT07846696
- 其他研究编号
- Nusinersen OLIP-02
日期
- 首次提交
- (20天前)
- 首次发布
- (昨天)
- 主要完成日期
- (2年后)
- 研究完成日期
- (2年后)
- 最近核实
- (29天前)
- 最近更新
- (昨天)
监管与共享
- FDA 监管药物
- 否
- FDA 监管器械
- 否
- 个体参与者数据共享计划
- UNDECIDED
- 是否有结果
- 否
We have no plans to publish this data.
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