An OLIP Study (Open Label Individual Patient) of Nusinersen in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen
2026年9月22日 更新者:Craig Campbell、London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
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
This study is an open label individual patient study of the drug Nusinsersen.
研究概览
详细说明
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.
研究类型
介入性
注册 (估计的)
1
阶段
- 不适用
联系人和位置
本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。
学习联系方式
- 姓名:Cara Grobbecker
- 电话号码:56216 519-685-8500
- 邮箱:cara.grobbecker@lhsc.on.ca
学习地点
-
-
Ontario
-
London、Ontario、加拿大、N6A 5W9
- Children's Hospital - London Health Sciences Centre
-
-
参与标准
研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 孩子
- 成人
- 年长者
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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 Spinal Muscular Atrophy 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 Spinal Muscular Atrophy [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.
学习计划
本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的:Open label
Open label |
Nusinersen intrathecal injection |
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Long-term safety and tolerability of Nusinersen 28mg dosing through adverse events experienced
大体时间:From enrollment to the end of treatment of one year
|
The descriptors of adverse events experienced |
From enrollment to the end of treatment of one year
|
合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (估计的)
2026年9月17日
初级完成 (估计的)
2028年12月31日
研究完成 (估计的)
2028年12月31日
研究注册日期
首次提交
2026年9月10日
首先提交符合 QC 标准的
2026年9月22日
首次发布 (实际的)
2026年9月24日
研究记录更新
最后更新发布 (实际的)
2026年9月24日
上次提交的符合 QC 标准的更新
2026年9月22日
最后验证
2026年9月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.