A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS) (CoMetS)
2026年7月30日 更新者：argenx
Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4-CMS
The purpose of this study is to assess efficacy and safety of adimanebart in participants at least 12 years of age with DOK7-, MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS). The study aims to determine whether adimanebart is safe and can help people with CMS feel better and perform daily activities more easily.
The study includes a double-blinded treatment period (DBTP) and an Open- label extension period (OLE). In the DBTP, all participants will be randomized in a 1:1 ratio to adimanebart or placebo. Participants who complete the DBTP will continue to the OLE.
Additionally, participants who complete part of the active-treatment period of ARGX-119-2302 study are eligible to enroll in the OLE of this study. In the OLE, all participants will receive open-label adimanebart. After final IMP dose, the participants will enter a follow-up period and their health will be monitored.
The total duration of the study is up to approximately 152 weeks (2 years and 11 months).
More information can be found here: clinicaltrials.argenx.com/Comets
研究概览
研究类型
介入性
注册 (估计的)
105
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
- 姓名：Sabine Coppieters, MD
- 电话号码：857-350-4834
- 邮箱：clinicaltrials@argenx.com
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
描述
Inclusion Criteria:
DBTP:
- At least 12 years of age.
- Has a diagnosis of DOK7-, MUSK-, AGRN-, or LRP4-CMS with documented mutations.
- Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) or other CMS medication must have been receiving the medication for at least 6 months and agree to remain on a same stable dosing regimen of the same medication unless directed to change their CMS medication(s) by their treating physician.
OLE:
- Completed part of the active-treatment period of ARGX-119-2302.
Exclusion Criteria:
DBTP:
- Known medical condition that would interfere with an accurate assessment of CMS, confound the results of the study, or put the patient at undue risk, as assessed by the investigator.
OLE:
- Investigational study drug discontinuation in ARGX-119-2302.
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：随机化
- 介入模型：并行分配
- 屏蔽：四人间
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的：Double-blinded treatment period (DBTP) - Adimanebart IV
Participants randomized to receive Adimanebart IV
|
Intravenous infusion of Adimanebart
|
|
安慰剂比较：Double-blinded treatment period (DBTP) - Placebo IV
Participants randomized to receive Placebo IV
|
Intravenous infusion of Placebo
|
|
实验性的：Open-label extension (OLE) - Adimanebart IV
Participants receive Adimanebart IV
|
Intravenous infusion of Adimanebart
|
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 at week 24 in 6MWT distance
大体时间：Up to 24 weeks
|
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
|
Up to 24 weeks
|
|
Incidence of AEs and SAEs
大体时间：up to 104 weeks
|
AE: adverse event; SAE: serious adverse event
|
up to 104 weeks
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Change from baseline in 6MWT distance over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in PROMIS PF-10b T-score over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The PROMIS PF-10b T-score (Patient-Reported Outcomes Measurement Information System Physical Function 10b) is a 10- item, participant-reported short-form questionnaire designed to assess physical function.
The questionnaire asks the participant to rate the items on a 5-point Likert scale of 5 (without any difficulty) to 1 (unable to do)
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in QMG key component composite score over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The Quantitative Myasthenia Gravis (QMG) scale is a standardized quantitative scoring system that was developed to assess disease severity based on impairment of body function and structures in patients with MG.
Minimum value: 0 (no disease severity); Maximum value: 15 (highest disease severity).
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in 6MWT cadence over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
Cadence is the number of steps taken per unit of time (steps/min) and is a measure of functional mobility.
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in the QMG key component raw values and scores over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The Quantitative Myasthenia Gravis (QMG) scale is a standardized quantitative scoring system that was developed to assess disease severity based on impairment of body function and structures in patients with MG.
Minimum value: 0 (no disease severity); Maximum value: 15 (highest disease severity).
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in PROMIS PF-WMA-SF T-score over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The PROMIS PF-WMA-SF T-score (Patient-Reported Outcomes Measurement Information System Physical Function With Mobility Aid Short Form) is an 11-item, participant-completed questionnaire that assesses lower and upper extremity function and associated activities of daily living.
The questionnaire asks the participant to rate the items on a 5-point scale of 5 (without any difficulty) to 1 (unable to do).
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in Neuro-QoL Short Form-Fatigue T-score over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
Neuro-QoL Short Form-Fatigue (Quality of Life in Neurological Disorders Short Form - Fatigue) is a participant-completed short-form questionnaire designed to provide a measurement of fatigue in patients with neurological conditions and the impact of their fatigue on their quality of life and daily activities.
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in FVC over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
FVC: forced vital capacity to assess pulmonary function
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in the Actigraphy measures over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
Participants will be asked to wear a wrist-worn actigraphy device to assess physical behaviour and mobility.
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in PGI-C over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The Patient Global Impression of Change (PGI-C) is a patient-reported rating scale to assess the change in symptom severity and functional problems since the first IMP administration.
Participants are asked to rate their change in symptom severity and functional problems on a 7- point Likert scale from 1 (much improved) to 7 (much worse).
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in PGI-S over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The Patient Global Impression of Severity (PGI-S) scale is a patient-reported rating scale to assess symptom severity and functional problems.
Participants are asked to rate their symptom severity and functional problems on a 7-point Likert scale from 1 (no problems) to 7 (unable to do).
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in CGI-C over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The Clinical Global Impression of Change (CGI-C) scale is a clinician-reported rating scale to assess changes in a participant's symptom severity and functional problems since the first IMP administration.
Clinicians are asked to rate the change in the participant's symptom severity and functional problems on a 7-point Likert scale from 1 (much improved) to 7 (much worse)
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in CGI-S over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The Clinical Global Impression of Severity (CGI-S scale) is a clinician-reported rating scale to assess a participant's symptom severity and functional problems.
The scale comprises a single item.
Based on clinical judgment, clinicians rate the participant's symptom severity and functional problems on a 4-point Likert scale from 0 (no problems) to 4 (unable to do).
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Change from baseline in EQ-5D-5L over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
The participant-completed EQ-5D-5L questionnaire is a standardized test recognized by many health authorities as a generic measure of health status.
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Incidence of AEs and SAEs
大体时间：up to 24 weeks
|
AE: adverse event; SAE: serious adverse event
|
up to 24 weeks
|
|
Adimanebart serum concentrations over time
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
PK=pharmacokinetic(s)
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Incidence of ADA against adimanebart
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
ADA: Anti-drug antibodies
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
|
Incidence of NAb against adimanebart
大体时间：up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
Nab: neutralizing antibody(ies)
|
up to 24 weeks (DBTP) + up to 104 weeks (OLE)
|
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
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1日
初级完成 (估计的)
2030年10月1日
研究完成 (估计的)
2030年10月1日
研究注册日期
首次提交
2026年7月30日
首先提交符合 QC 标准的
2026年7月30日
首次发布 (实际的)
2026年8月4日
研究记录更新
最后更新发布 (实际的)
2026年8月4日
上次提交的符合 QC 标准的更新
2026年7月30日
最后验证
2026年7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
- ARGX-119-17-CMS-3001
- 2025-524460-37-00 (克蒂斯)
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
不
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
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