A Phase 1b, Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- argenx
- 入组人数
- 15
- 试验地点
- 16
- 主要终点
- Assessment of adverse events (AEs)
研究概览
简要总结
The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function.
After the screening period, eligible participants will be randomized in a 4:1 ratio to receive intravenous infusions of ARGX-119 or placebo during the double-blinded treatment period. Participants will then enter the follow-up period. After the follow-up period, participants may enrol in the active-treatment period, where they will receive open-label ARGX-119.
The full duration of the study is approximately 38 months.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At least 18 years of age.
- •Has genetically confirmed congenital myasthenic syndromes due to mutation of downstream of kinase 7 (DOK7-CMS).
- •Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) must have been receiving the medication for more than 3 months and agree to remain on a same stable dosing regimen of the same medication until the end of the study.
排除标准
- •Diagnosis of CMS due to mutation of any gene other than DOK
- •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.
- •History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for more than 5 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer.
- •Pregnant or lactating state or intention to become pregnant during the study.
研究组 & 干预措施
Double-blinded treatment period - Placebo IV
Participants receive placebo during the double-blinded treatment period
干预措施: Placebo (Other)
Double-blinded treatment period - ARGX-119 IV
Participants receive ARGX-119 during the double-blinded treatment period
干预措施: ARGX-119 (Biological)
Active-treatment period - ARGX-119 IV
Participants receive ARGX-119 during the active-treatment period
干预措施: ARGX-119 (Biological)
结局指标
主要结局
Assessment of adverse events (AEs)
时间窗: Up to week 42
Change from active-treatment baseline over time for 6MWT distance
时间窗: Up to 72 weeks
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes. Before and after the 6MWT assessment, the participant's blood pressure, heart rate, and SPO2 will be recorded, and the participant's perception of fatigue and dyspnea will be measured.
次要结局
- Maximum observed serum concentration (Cmax) of ARGX-119(Up to 42 weeks + 72 weeks)
- Incidence of ADA against ARGX-119(Up to 42 weeks + 72 weeks)
- Change from baseline over time for key components of the QMG scale(Up to 42 weeks + 72 weeks)
- Change from baseline over time for MG-ADL(Up to 42 weeks + 72 weeks)
- Change from baseline over time for PROMIS-GH scale(Up to 42 weeks)
- Change from active-treatment baseline over time for 6MWT cadence(Up to 72 weeks)
- Change from active-treatment baseline over time for PROMIS PF-WMA-SF(Up to 72 weeks)
- Change from active-treatment baseline over time for Neuro-QoL fatigue(Up to 72 weeks)
- Change from active-treatment baseline over time for FVC(Up to 72 weeks)
- Change from active-treatment baseline over time for PGI-C(Up to 72 weeks)
- Change from active-treatment baseline over time for PGI-S(Up to 72 weeks)
- Change from active-treatment baseline over time for CGI-C(Up to 72 weeks)
- Change from active-treatment baseline over time for CGI-S(Up to 72 weeks)
- Change from active-treatment baseline over time for EQ-5D-5L(Up to 72 weeks)
- Incidence of AEs and SAEs(Up to 72 weeks)