此页面是自动翻译的，不保证翻译的准确性。请参阅 英文版 对于源文本。

一项关于Salanersen（BIIB115）对15至60岁脊髓性肌萎缩症（SMA）患者运动功能影响及安全性的研究，研究对象包括SMA治疗初治患者和既往接受过Risdiplam治疗的患者 (SOLAR)

2026年9月29日 更新者：Biogen

一项开放标签、III 期研究，旨在评估 Salanersen (BIIB115) 在 15-60 岁脊髓性肌萎缩症参与者中的疗效和安全性，这些参与者既往未接受过治疗或曾接受过 Risdiplam 治疗

In this study, researchers will learn more about the effects and safety of BIIB115, also known as salanersen.

Specifically, researchers will learn more about how salanersen works in individuals with SMA who are between the ages of 15 and 60 years old. In most people living with SMA, changes to or a lack of a gene called survival motor neuron 1 (SMN1) - often referred to as gene mutations or variants - affect how this gene works. As a result, their bodies produce less SMN protein. Without enough of this protein, motor neurons and muscles cannot work properly. There is a similar gene called SMN2 that produces SMN protein, but it usually does not produce enough SMN protein on its own to make up for the changes in the SMN1 gene. Salanersen is a drug designed to help the SMN2 gene to make more working SMN protein.

In this study, there will be 2 groups of participants: a group who has never received treatment for SMA before joining this study, and a group who has been treated with risdiplam, an approved drug for SMA . Those participants must not have received any other SMA treatments before and will need to stop their risdiplam treatment for the duration of the study.

The main goal of this study is to learn more about how salanersen affects the participants' motor function. Researchers will use different tests and questionnaires to learn if motor function is changing over the study duration.

The main question researchers want to answer in this study is:

• For the group who has never been treated for SMA, how much do scores on the HFMSE movement test change at 12 months compared to the beginning of the study? The Hammersmith Functional Motor Scale - Expanded (HFMSE) has 33 activities that are scored which include sitting, lying down, walking, jumping, and more.

Researchers will also learn more about:

  • The effects on participants' motor function and how well their nerves and muscles function.
  • The effects on participants' overall sense of change and how they perform daily activities.
  • How many participants have adverse events or serious adverse events. Adverse events are health problems that may or may not be caused by the study drug.
  • How much salanersen gets into the fluid surrounding the brain and spinal cord.
  • How much salanersen gets into the blood.

This study will be done as follows:

  • First, participants will be screened to check if they can join the study. The screening period may be up to 4 weeks.
  • This is an "open-label" study. This is a study in which the participants, study doctor, and site staff will know that participants are receiving salanersen.
  • All participants will receive salanersen through an intrathecal injection, or one that is given into the fluid surrounding the brain and spinal cord.
  • Participants will receive salanersen once every year for a total of 5 times throughout the study.
  • Including screening, participants will have 17 study visits and 9 telephone calls during this study, which will last up to 61 months in total.

研究概览

地位

招聘中

条件

干预/治疗

详细说明

The primary objective of the SOLAR study is to evaluate the clinical efficacy of salanersen in participants with SMA who are treatment-naïve or previously treated with risdiplam. The secondary objective of the study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of salanersen.

研究类型

介入性

注册 (估计的)

90

阶段

  • 第三阶段

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

研究联系人备份

学习地点

  • 中国
      • Beijing、中国、100730
        • 招聘中
        • Peking Union Medical College Hospital
  • 美国
    • California
      • Palo Alto、California、美国、94304
        • 招聘中
        • Stanford University Medical Center
        • 接触：
          • 电话号码：650-725-7622
        • 首席研究员：
          • John Day
    • Virginia
      • Norfolk、Virginia、美国、23507
        • 招聘中
        • Childrens Hospital of the Kings Daughter Norfolk
        • 首席研究员：
          • Crystal Proud
        • 接触：
          • 电话号码：757-668-6981

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

15年 至 60年 (孩子、成人)

接受健康志愿者

不

有资格学习的性别

全部

描述

Key Inclusion Criteria:

  • Participants aged 15 to 60 years, inclusive, at the time of informed consent

  • Participants with genetic documentation of 5q Spinal Muscular Atrophy (SMA) (homozygous gene deletion or mutation or compound heterozygous mutation).

  • Participants with clinical signs and symptoms consistent with SMA.

  • Survival motor neuron 2 (SMN2) copy number ≥ 1.

  • Participants with baseline Hammersmith Functional Motor Scale - Expanded (HFMSE) total score of ≥ 10 to ≤ 54.

  • Participants who are able to sit without using support for at least 10 seconds.

  • Participants with no prior treatment with myostatin inhibitors and a willingness to remain off concurrent myostatin inhibitor therapy for the duration of the study.

  • Ambulatory and nonambulatory participants:

    • Ambulatory participants must be able to walk at least 10 meters independently without assistance and are willing and able to complete the 6 Minute Walk Test (6MWT) at Screening.

  • For participants in the treatment-naïve cohort:

    • No prior treatment with an approved SMA Disease Modifying Therapy (DMT) or an investigational drug given for the treatment of SMA.

  • For participants in the risdiplam-treated cohort:

    • Currently receiving risdiplam treatment and have been on once-daily 5 milligrams (mg) risdiplam treatment for at least 6 months prior to Screening.
    • Willing to stop risdiplam therapy for the duration of the study. The last dose of risdiplam must be taken the day before the first dose of salanersen.
    • No prior treatment with nusinersen, onasemnogene abeparvovec-xioi/onasemnogene abeparvovec-brve (OA), other approved DMTs for SMA or investigational drugs given for the treatment of SMA apart from risdiplam.

Key Exclusion Criteria:

  • Respiratory insufficiency at Screening, defined by the medical necessity for invasive or noninvasive ventilation for > 6 hours during a 24-hour period (except for nocturnal bilevel positive airway pressure).
  • Medical necessity for a gastric feeding tube, where the majority of nutrition is provided by this route, as assessed by the site Investigator at Screening.
  • History of brain or spinal cord disease or other contraindications (e.g., severe scoliosis) that would interfere with the lumbar puncture (LP) procedures, Cerebrospinal fluid (CSF) circulation, efficacy assessments, or safety assessments (including a history of hydrocephalus or implanted shunt for CSF drainage), as assessed by the Investigator.
  • Hospitalization for surgery, a pulmonary event, or nutritional support within 2 months prior to Screening or plans to undergo elective procedures or surgeries at any time after signing the Informed Consent Form (ICF) through the end of the study. Note: If prior scoliosis surgery has been performed, it must be done at least 1 year prior to Screening.
  • Presence of an active medical issue (e.g., infection, recent fracture) that would make the participant unsuitable for inclusion, as assessed by the Investigator.
  • Current enrollment or a plan to enroll in any interventional clinical study in which an investigational treatment or approved therapy for investigational use is administered within 90 days or 5 half-lives of the treatment (if known), whichever is longer, prior to Screening. This includes neuromodulation therapy such as spinal cord stimulation.

Note: Other protocol-defined inclusion/exclusion criteria will apply.

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：并行分配
  • 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：Treatment-Naïve Cohort

Treatment-naïve participants will receive salanersen 80 milligrams (mg) by intrathecal (IT) lumbar puncture (LP) every 12 months for a total of five doses.
药品：Salanersen

Administered Intrathecally

其他名称：
  • BIIB115
实验性的：Risdiplam-Treated Cohort

Risdiplam-treated participants will receive salanersen 80 mg by IT LP every 12 months for a total of five doses.
药品：Salanersen

Administered Intrathecally

其他名称：
  • BIIB115

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
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Total Score in Treatment-Naïve Cohort
大体时间：At Month 12

The HFMSE is a tool used to assess motor function in individuals with SMA. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all 33 items, with a maximum score of 66 with higher scores depicting better ability to perform activities.
At Month 12

次要结果测量

结果测量
措施说明
大体时间
出现不良事件（AEs）和严重不良事件（SAEs）的参与者人数
大体时间：最长至第1825天
最长至第1825天
Salanersen 在脑脊液 (CSF) 中的浓度
大体时间：最长至第1460天
最长至第1460天
血清中Salanersen的浓度
大体时间：长达第1825天
长达第1825天
Percentage of Participants With ≥ 3-Point Change From Baseline in HFMSE Total Score
大体时间：Up to Day 1825

The HFMSE is a tool used to assess motor function in individuals with SMA. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all 33 items, with a maximum score of 66 with higher scores depicting better ability to perform activities.
Up to Day 1825
Percentage of Participants With ≥ 2-Point Change From Baseline in Revised Upper Limb Module (RULM) Total Score
大体时间：Up to Day 1825

The RULM is developed to assess upper limb functional abilities of participants with SMA. This test consists of a total of 20 upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function.
Up to Day 1825
Percentage of Participants With ≥ 30-Meter Change From Baseline in 6-Minute Walk Test (6MWT) Distance (Ambulatory Participants Only)
大体时间：Up to Day 1825

The 6MWT is a submaximal exercise test used to assess an individual's functional exercise capacity. It measures the distance covered in 6 minutes on a flat, hard surface, providing valuable information about aerobic capacity and endurance.
Up to Day 1825
Change From Baseline in HFMSE Total Score
大体时间：Up to Day 1825

The HFMSE is a tool used to assess motor function in individuals with SMA. Participants will be asked to complete a specific movement and are then graded on the quality and execution of that movement. Higher scores indicate higher levels of motor ability. The overall score is the sum of the scores for all 33 items, with a maximum score of 66 with higher scores depicting better ability to perform activities.
Up to Day 1825
Change From Baseline in RULM Total Score
大体时间：Up to Day 1825

The RULM is developed to assess upper limb functional abilities of participants with SMA. This test consists of a total of 20 upper limb performance items that are reflective of activities of daily living. The RULM is scored from 0 to 37 points, with higher scores indicating better function.
Up to Day 1825
Change From Baseline in Total 6MWT Distance (Ambulatory Participants Only)
大体时间：Up to Day 1825

The 6MWT is a submaximal exercise test used to assess an individual's functional exercise capacity. It measures the distance covered in 6 minutes on a flat, hard surface, providing valuable information about aerobic capacity and endurance.
Up to Day 1825
Change From Baseline in Compound Muscle Action Potential (CMAP) Amplitudes
大体时间：Up to Day 1825

CMAP is a well validated method for tracking disease progression in neuromuscular disorders such as SMA and amyotrophic lateral sclerosis and has been proposed as a potential biomarker of a therapeutic effect in SMA. CMAPs will be performed for the following nerve-muscle pairs: ulnar-abductor digiti minimi and peroneal-tibialis anterior.
Up to Day 1825
Patient Global Impression of Change (PGI-C) Score
大体时间：Up to Day 1825

PGI-C is a self-reported 7-point scale evaluating the participant's perception of change in disease state since baseline. The scale ranges from 1 to 7, where 1= very much improved; 2= much improved; 3= minimally improved; 4= no change; 5= minimally worse; 6= much worse; or 7= very much worse. Lower scores indicate clinical improvement, and higher scores indicate disease worsening.
Up to Day 1825
Change From Baseline in SMA Independence Scale - Upper Limb Module (SMAIS-ULM)
大体时间：Up to Day 1825

The SMAIS-ULM is a validated tool designed to assess the level of independence in daily activities related to upper limb functionality for individuals with Type II and nonambulant Type III SMA. The 22 items assess upper limb focused tasks across 5 domains of typical daily activities (bathing/hygiene, dressing, eating/drinking, picking up/moving objects, and other tasks) scored on a 3-point scale (0 = I cannot do this at all without help; 1 = I need some help; and 2 = I do not need help). Higher scores indicate greater independence.
Up to Day 1825

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。

赞助

Biogen

调查人员

  • 研究主任：Medical Director、Biogen

出版物和有用的链接

负责输入研究信息的人员自愿提供这些出版物。这些可能与研究有关。

有用的网址

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 (实际的)

2026年9月8日

初级完成 (估计的)

2028年6月23日

研究完成 (估计的)

2032年6月22日

研究注册日期

首次提交

2026年2月26日

首先提交符合 QC 标准的

2026年2月26日

首次发布 (实际的)

2026年3月3日

研究记录更新

最后更新发布 (实际的)

2026年10月2日

上次提交的符合 QC 标准的更新

2026年9月29日

最后验证

2026年9月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • 277SM303
  • 2025-524054-34 (其他标识符：EU Trial (CTIS) Number)
  • 2025 (美国 NIH 拨款/合同)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

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.

订阅