NCT05225675已完成2 期
A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, Multicenter Trial to Evaluate the Safety and Tolerability, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 2 Dose Regimens of ARGX-117 in Adults With Multifocal Motor Neuropathy
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- argenx
- 入组人数
- 54
- 试验地点
- 79
- 主要终点
- Safety outcomes based on adverse event (AE) monitoring and other safety assessments
研究概览
简要总结
This is a phase 2, randomized, double-blinded, placebo-controlled, parallel-group, multicenter trial to evaluate the safety and efficacy of 2 dose regimens of ARGX-117 versus placebo, in participants with MMN previously stabilized with IVIg (intravenous immunoglobulin).
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
- •Capable of giving signed informed consent form (ICF)
- •Male/female at least 18 years of age at the time the informed consent form (ICF) is signed
- •Probable or definite MMN according to the European Federation of Neurological Societies (EFNS)/Peripheral Nerve Society (PNS) (EFNS/PNS) 2010 guidelines at screening confirmed by the MMN Confirmation Committee (MCC)
- •Receiving a stable IVIg regimen for at least 3 months before screening or recently initiated IVIg treatment
- •IVIg treatment dependency confirmation by the MMN Confirmation Committee (MCC)
- •Immunization with the first meningococcal vaccine and pneumococcal vaccine, and the single Haemophilus influenza type B vaccine must be performed at least 14 days before IMP administration at V1 according to local country-specific immunization schedules. A documented history of vaccination against Neisseria meningitides, Haemophilus influenza type B, and streptococcus pneumonia will be permitted
- •Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
排除标准
- •Any coexisting condition which may interfere with the outcome assessments
- •Clinical signs or symptoms suggestive for neuropathies other than MMN such as motor neuron disease or other inflammatory neuropathies
- •Severe psychiatric disorder, history of suicide attempt, or current suicidal ideation that in the opinion of the investigator could create undue risk to the participant or could affect adherence with the trial protocol.
- •Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection during the screening and/or IVIg monitoring period (IVMP).
- •Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of MMN or put the participant at undue risk (eg, SLE).
- •History of malignancy unless resolved by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Participants with the following carcinomas will be eligible:
- •Adequately treated basal cell or squamous cell skin cancer
- •Carcinoma in situ of the cervix
- •Carcinoma in situ of the breast
- •Incidental histological finding of prostate cancer
- •Clinical evidence of other significant serious diseases, have had a recent major surgery (including a splenectomy at any time), or who have any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk
- •Prior/concomitant therapy
- •Cyclophosphamide and/or rituximab and/or eculizumab and/or mycophenolate mofetil within 3 months prior to screening
- •Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP.
- •Positive serum test at screening for an active viral infection with any of the following conditions:
- •Hepatitis B virus (HBV) that is indicative of an acute or chronic infection
- •Hepatitis C virus (HCV) based on HCV antibody assay
- •HIV based on test results that are associated with an AIDS-defining condition
- •Current or history of (ie, within 12 months of screening) alcohol, drug, or medication abuse
- •Known hypersensitivity reaction to 1 of the components of the IMP or any of its excipients
- •Female participants with a positive serum or urine pregnancy test, lactating females, and those who intend to become pregnant during the trial or within 15 months after last dose of the IMP
- •ALT or AST ≥2 × upper limit of normal and total bilirubin ≥1.5 × upper limit of normal of the central laboratory reference range
- •An estimated glomerular filtration rate of ≤60 mL/min/1.73m2
研究组 & 干预措施
Placebo
Placebo Comparator
Intravenous administration of placebo
干预措施: Placebo (Other)
ARGX-117
Experimental
Intravenous administration of ARGX-117
干预措施: ARGX-117 (Biological)
结局指标
主要结局
Safety outcomes based on adverse event (AE) monitoring and other safety assessments
时间窗: 16 weeks
次要结局
- AUC (area under curve) of the change from baseline in mMRC (modified Medical Research Council)-10 sum score(16 weeks)
- Value baseline in the mMRC (modified Medical Research Council)-14 sum score(16 weeks)
- Proportion of participants showing a deterioration of 1 or more points in at least 2 muscle groups as assessed by the mMRC (modified Medical Research Council)-14 sum score(16 weeks)
- Proportion of participants with no deterioration in 2 or more muscle groups as assessed by mMRC (modified Medical Research Council)-14 sum score(16 weeks)
- Values baseline in GS (grip strength)(16 weeks)
- Percent change from baseline in GS (grip strength)(16 weeks)
- Time to the first retreatment with IVIg since the final IVIg treatment of the IVIg monitoring period(16 weeks)
- Change from baseline in the average score of the 2 most important muscle groups as assessed by the mMRC (modified Medical Research Council)-14 sum score(16 weeks)
- Proportion of participants with a GS (grip strength) decrease of 8 kilopascal (kPa) or more over 3 consecutive days(16 weeks)
- Change from baseline in GS (grip strength)(16 weeks)
- Change from baseline in the mMRC (modified Medical Research Council)-14 sum score(16 weeks)
- Values baseline in the average time for upper extremity (arm and hand) function (9-Hole Peg Test [9-HPT], or timed Peg Board Test)(16 weeks)
- Change from baseline in the average time for upper extremity (arm and hand) function (9-Hole Peg Test [9-HPT], or timed Peg Board Test)(16 weeks)
- Serum titer levels of binding antibodies (BAbs) against ARGX-117(16 weeks)
- Maximum serum concentrations (Cmax)(16 weeks)
- AUC (area under curve) of the change from baseline in GS (grip strength)(16 weeks)
- Change from baseline in quality of life using EQ-5D-5L visual analog scale(16 weeks)
- Change from baseline in the chronic acquired polyneuropathy patient-reported index (CAP-PRI)(16 weeks)
- Values baseline in free C2, total C2, functional complement activity (CH50)(16 weeks)
- Values baseline in the Rasch-built overall disability scale for MMN (MMN-RODS©)(16 weeks)
- Change from baseline in the Rasch-built overall disability scale for MMN (MMN-RODS©)(16 weeks)
- Proportion of participants by level of severity on each dimension of the EQ-5D-5L scale(16 weeks)
- Change from baseline in free C2, total C2, functional complement activity (CH50)(16 weeks)
- Area Under The Curve (AUC)(16 weeks)
研究者
研究点 (79)
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