Efficacy and Safety of Subcutaneous Immunoglobulin in Patients With Myasthenia Gravis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Myasthenia Gravis Impairment Index Efficacy Outcome
研究概览
简要总结
This is a prospective open-label, randomized, parallel arm clinical trial.
The primary objective of the study is to evaluate the safety and efficacy of Cuvitru 20% subcutaneous immunoglobulin in patients with myasthenia gravis (MG). The secondary objective is to evaluate patient preferences and effects on quality of life when treating MG patients with SCIG. Exploratory objectives are to compare de novo administration starting SCIG directly with those starting with a loading dose of IVIG followed by SCIG administration.
Patients over age 18 with moderate to severe MG with MGFA Class II-IV without contraindications to immunoglobulin will be considered for the study.
All patients will be eligible to enter either arm of the study, Arm 1: 10% Gammagard IVIG followed by 20% Cuvitry SCIG and Arm 2: Cuvitru 20% SCIG alone.
详细描述
Myasthenia gravis (MG) is an autoimmune neuromuscular condition which can cause fatigable weakness of skeletal muscles including bulbar, ocular, limb, axial and respiratory muscles. Symptoms range from mild, transient double vision and ptosis to severe, life threatening diffuse weakness, and treatments which are immunosuppressive or immunomodulatory have been shown to improve outcome in MG. This includes 10% intravenous immunoglobulin (IVIG), which has previous been shown by our group to improve strength at 2 and 4 weeks in patients with MG. Due to delay in the onset of many immunosuppressives and potential side effects from steroids and other immunosuppressive medications, IVIG is also used in clinical practice as bridging of maintenance therapy until other therapies take effect or the patient stabilizes. In addition, there have been other studies suggesting that 20% subcutaneous immunoglobuin (SCIG) can be helpful to improve strength in patients with MG over a relatively short period of time (6 weeks). Some of these studies are ongoing and are further evaluating the ability of IVIG and SCIG to stabilize or improve strength IVIG in patients with MG, however, there is no current published study evaluating different administration routes of immunoglobulin (IVIG and SCIG) in patients with MG over a longer 6 month follow-up period.
The current study aims to be the first to evaluate 20% Cuvitru SCIG formulation in patients with neuromuscular conditions including MG. The study aim is to evaluate the safety and efficacy of 6 months of 20% SCIG Cuvitru treatment vs three months of 10% IVIG "pre-treatment" followed by 3 months of 20% SCIG Cuvitru treatment. The study will also be the first to use the myasthenia gravis impairment index (MGII) as primary outcome in conjunction with standard MG outcomes.
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
- None
入排标准
- 年龄范围
- 18 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients over age 18
- •Patients with a confirmed diagnosis of myasthenia gravis based on clinical criteria including fatiguable weakness and supported by either serological (acetylcholine receptor, muscle specific kinase or anti-low-density lipoprotein receptor- related protein 4 antibodies or electrophysiological testing (repetitive nerve stimulation of single fiber electromyography)
- •Myasthenia Gravis Federation of America class II-IV
- •Moderate to severe myasthenia gravis as defined by a quantitative myasthenia gravis score >10 or generalized myasthenia gravis impairment index score > 11
- •Patient able to give consent and is able and willing to complete all study procedures and activities
排除标准
- •Patients who are pregnant or breastfeeding
- •Patients not able to complete the study procedures or with an alternate diagnosis
- •Patients with recent thymectomy in the past 6 months
- •Patients receiving another biologic agent such as rituximab, belimubab, cyclophosphamide and eculizumab in the past 6 months prior to study entry
- •No IVIG or subcutaneous immunoglobulin within the past month
- •Patients on prednisone who have had alterations in prednisone dose over the past month prior to study entry
- •Patients will previous known allergy or severe adverse reaction to intravenous or subcutaneous immunoglobulin
- •Evidence of renal insufficiency (Cr>1.5 x elevated) or liver disease (transaminases > 2.5 x elevation) at screening.
研究组 & 干预措施
IVIG + SCIG
This group of MG patients will start with 2g/kg of IVIG on month 1, 1 g/kg of IVIG 4 and then 8 weeks later, and within 2 weeks switch to SCIG treatment
干预措施: intravenous immunoglobulin + subcutaneous immunoglobulin (SCIG) (Drug)
SCIG alone
This group of MG patients will start with SCIG alone
干预措施: subcutaneous immunoglobulin (SCIG) (Drug)
结局指标
主要结局
Myasthenia Gravis Impairment Index Efficacy Outcome
时间窗: 6 months
Change in the Myasthenia Gravis Impairment Index (score 0-84, with 84 being the maximal score indicating the most severe MG status) at baseline compared to end of study
次要结局
- Myasthenia Gravis Composite Efficacy Outcome(6 months)
- Myasthenia Gravis Activities of Daily Living Efficacy Outcome(6 months)
- Quantitative Myasthenia Gravis Score Efficacy Outcome(6 months)
- Myasthenia gravis Quality of Life 15 Score Efficacy Outcome(6 months)
- Parallel Arm Comparison(6 months)
- Safety and Tolerability: total number of adverse events(6 months)
研究者
Hans Katzberg
Neurologist, Principle Investigator
University Health Network, Toronto