2024-516473-72-00招募中3 期
A Phase 3, Randomized, Double-Blinded, Double-Dummy Study Evaluating the Efficacy and Safety of Empasiprubart Versus Intravenous Immunoglobulin in Adults With Multifocal Motor Neuropathy.
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Argenx
- 入组人数
- 134
- 试验地点
- 70
- 主要终点
- Change from baseline in GS (3 day moving average) in the most affected hand at week 24
研究概览
简要总结
Part A: To demonstrate the efficacy of empasiprubart compared to IVIg in improving muscle strength
研究设计
- 分配方式
- Non-randomized
- 主要目的
- Part B
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Is at least 18 years of age and the local legal age of consent for clinical studies
- •Has a confirmed diagnosis of definite or probable MMN at screening according to the EFNS/PNS 2010 guidelines
- •Has responded to IVIg in the past 5 years.
- •Is receiving IVIg at a treatment interval of once every 2, 3, 4, or 5 weeks, and a dose of 0.4 to 2.0 g/kg body weight per cycle
- •Is receiving a maintenance regimen (no change in frequency, and no change in dose >10%) of IVIg for at least 8 weeks before screening (or at least 10 weeks for participants receiving IVIg once every 5 weeks)
- •Minimum converted weekly IVIg dose of ≥0.125 g/kg
- •Has documented immunization against encapsulated bacterial pathogens (N meningitidis and S pneumoniae) within 5 years of screening or is willing to receive immunization at least 14 days before first study drug administration
排除标准
- •Besides the indication under study, known autoimmune disease (eg, SLE) or any other medical condition that would confound the study results or put the participant at undue risk
- •Clinical signs or symptoms suggestive of neuropathies other than MMN, such as motor neuron disease (eg, bulbar signs, brisk reflexes) or other inflammatory neuropathies (eg, sensory neuropathy)
结局指标
主要结局
Change from baseline in GS (3 day moving average) in the most affected hand at week 24
Change from baseline in GS (3 day moving average) in the most affected hand at week 24
次要结局
- Part A - Change from baseline in MMN-RODS centile score at week 24
- Part A - Change from baseline in mMRC-14 sum score at week 24
- Part A - PGI-C actual value over time
- Part A - Change from baseline in CAP-PRI total score over time
- Part A - Percentage change from baseline in time to complete the 9-HPT with the dominant hand at week 24
- Part A - Incidence and severity of AEs and AESIs, Incidence of SAEs
- Part A - Clinically meaningful changes in laboratory parameters, vital signs, and ECG results
- Part A - Serum concentrations over time and PK parameters of empasiprubart
- Part A - Values and percentage change from baseline in free C2 and total C2 over time
- Part A - Incidence and prevalence of anti-drug antibodies (ADA) against empasiprubart in serum
- Part A - Incidence and prevalence of NAb against empasiprubart in serum
- Part A - Change from baseline in GS (3-day moving average) of the least affected hand over time and AUC of change from baseline in GS (daily average) for both hands; percentage change from baseline in GS (3-day moving average) for both hands
- Part A - Percentage change from baseline in time to complete the 9-HPT with the nondominant hand over time
- Part A - Change from baseline in sum scores for mMRC-10 and mMRC-14 restricted to the 2 most affected muscle groups over time
- Part A - Proportion of participants and shift from baseline over time by level of severity on PGI-S
- Part A - Change from baseline in Rasch-Transformed Fatigue Severity Scale (RT-FSS) score over time
- Part A - Change from baseline in physical component and mental component scores of 12-Item Short Form Survey (SF-12) over time
- Part A - Proportion of participants and shift from baseline by each dimension of the EQ5D-5L scale, change from baseline in the EQ-5D-5L visual analog scale over time, and change from baseline in EQ-5D-5L valuation index
- Part B - Actual values of and changes from baseline in MMN-RODS centile score, CAPPRI total score, grip strength (daily average; both hands), mMRC-10 sum score, mMRC-14 sum score, and mMRC-14 sum score restricted to the 2 most affected muscle groups over time
- Part B - Actual values of and percentage change from baseline in time to complete the 9-HPT with the dominant and nondominant hands over time
- Part B - Actual values of PGI-C and PGI-S over time
- Part B - Incidence and severity of AEs and AESIs, Incidence of SAEs
- Part B - Clinically meaningful changes in laboratory parameters, vital signs, and ECG results
- Part B - Serum concentrations over time and PK parameters of empasiprubart
- Part B - Values and percentage change from baseline in free C2 and total C2 over time
- Part B - Incidence and prevalence of ADA against empasiprubart in serum
- Part B - Incidence and prevalence of NAb against empasiprubart in serum
- Part B - Change from baseline in RT-FSS score over time
- Part B - Change from baseline in physical component and mental component scores of SF-12 over time
- Part B - Proportion of participants and shift from baseline by each dimension of the EQ5D-5L scale and change from baseline in the EQ-5D-5L visual analog scale over time
研究者
Chief Scientific Officer
Scientific
Argenx
研究点 (70)
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