Efgartigimod in IVIG Dependent Myasthenia Gravis Patients
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- MG-ADL
研究概览
简要总结
This study is an open label, single center, prospective, 26 weeks study with descriptive analysis where IVIG is replaced by efgartigimod therapy. MG-ADL and MGQOL evaluations will occur weekly throughout the study to week 26.
详细描述
Objectives:
To determine if efgartigimod is equivalent or non-inferior in efficacy to IVIG in the treatment of stable, acetylcholine receptor antibody positive, IVIG dependent myasthenia gravis patients.
To determine patient treatment preference between IVIG and efgartigimod in stable, IVIG dependent acetylcholine receptor antibody positive myasthenia gravis patients.
To determine the safety and tolerability of efgartigimod administered in a fixed regular dosing regimen over 6 months in stable IVIG dependent acetylcholine receptor antibody positive myasthenia gravis patients.
IVIG will be discontinued one week prior to the baseline visit (week 0) while other concurrent medications (non-steroidal immunosuppressive therapies (NSISTs), acetylcholinesterase (AChE) inhibitors or corticosteroids) for myasthenia will be kept unchanged for the duration of the study. The dose and frequency of efgartigimod treatment will be 10mg/kg (max of 1200mg) administered as a one-hour intravenous infusion every week x 4 infusions followed by a four-week break and repeated for a total four treatment cycles till week 24 and then followed by a two-week observation with an end of study visit at week 26.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed informed consent.
- •Age 18-80 years
- •Acetylcholine receptor antibody positive, myasthenia gravis patients, with stable disease for the past four or more months. Stable disease is defined as no change in dosage or interval in IVIG treatments and without any significant change in clinical status.
- •No modification or addition of NSISTs in the past six months
- •No modification or addition in corticosteroid therapy for the past three months
- •Myasthenia Gravis diagnosis was supported by abnormal neurotransmission test or history of improvement with AChE inhibitors.
- •Receiving chronic regular IVIG treatments for myasthenia gravis for the past year or more.
排除标准
- •Patients with previous rituxan or eculizumab treatment or plasma exchange within the past six months
- •Patients with previous thymectomy within the past 3months
- •Patients that have active Hepatitis B, are seropositive for Hepatitis C or HIV or have latent, untreated or active TB or any other significant active infection
- •Patients that have at screening a serum IgG less than 6.0gm/L or a history of chronic hypogammaglobulinemia from any cause.
- •Patients that are pregnant or considering becoming pregnant in the next 6 months.
- •Patients with severe renal impairment (eGFR less than 30ml/min)
- •Patients who in the opinion of the investigator should not participate in the study.
研究组 & 干预措施
treatment
Efgartigimod infusion
干预措施: efgartigimod administration (Drug)
结局指标
主要结局
MG-ADL
时间窗: a sustained decrease persistent for at least 4 consecutive weeks over the 26 week period
The percentage of patients who do not experience, at any time during the study, a 2 or more-point deterioration from their averaged baseline MG-ADL score that is sustained for a period of 4 or more consecutive weeks. (stable or improved patients)
次要结局
- QMG(those who experience a sustained decrease for 4 consecutive weeks during the 26 week period)
- MGQOL(during the whole study period of 26 weeks)
- incidence of patients who are still receiving efgartigimod by the end of the 26 weeks(for the duration of the 26 week study)
研究者
Dr. Francois Jacques
Neurologist
Clinique Neuro-Outaouais