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
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 发起方
- argenx
- 入组人数
- 105
- 主要终点
- Change from baseline at week 24 in 6MWT distance
研究概览
简要总结
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
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
- •Completed part of the active-treatment period of ARGX-119-2302.
排除标准
- •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.
- •Investigational study drug discontinuation in ARGX-119-2302.
结局指标
主要结局
Change from baseline at week 24 in 6MWT distance
时间窗: Up to 24 weeks
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes.
Incidence of AEs and SAEs
时间窗: up to 104 weeks
AE: adverse event; SAE: serious adverse event
次要结局
- Change from baseline in 6MWT distance over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in PROMIS PF-10b T-score over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in QMG key component composite score over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in 6MWT cadence over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in the QMG key component raw values and scores over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in PROMIS PF-WMA-SF T-score over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in Neuro-QoL Short Form-Fatigue T-score over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in FVC over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in the Actigraphy measures over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in PGI-C over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in PGI-S over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in CGI-C over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in CGI-S over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Change from baseline in EQ-5D-5L over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Incidence of AEs and SAEs(up to 24 weeks)
- Adimanebart serum concentrations over time(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Incidence of ADA against adimanebart(up to 24 weeks (DBTP) + up to 104 weeks (OLE))
- Incidence of NAb against adimanebart(up to 24 weeks (DBTP) + up to 104 weeks (OLE))