A Pilot Trial To Assess The Feasibility And Efficacy Of Subcutaneous Immunoglobulin In Patients With Myasthenia Gravis Exacerbation
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 26
- 试验地点
- 2
- 主要终点
- Change in Quantitative Myasthenia Gravis Score (QMGS) from baseline to day 42 after treatment.
研究概览
简要总结
This is a prospective open-label, uncontrolled, single-blind, pilot clinical trial.
The primary objective is to assess the efficacy, safety, feasibility and tolerability of SCIG in patients with worsening MG.
Participants with moderate worsening of MG symptoms (MGFA Class II and III) who are considered to be appropriate for immunoglobulin therapy will be screened for the study by the treating neurologist.
Patients will be receive 2gm/kg (150gm for a 75kg patient) of 20% SCIG (Hizentra) infused over 4 weeks in a dose escalating manner.
Additionally, this study will be assessing the feasibility of employing SCIG as an alternative therapy to IVIG in patients with MG exacerbation. The cost-effectiveness of SCIG versus IVIG will be evaluated, and the impact of SCIG therapy will be assessed from both a health-resource perspective and from a patient perspective.
详细描述
This is a prospective open-label, uncontrolled, single-blind, pilot clinical trial.
The primary objective is to assess the efficacy, safety, feasibility and tolerability of SCIG in patients with worsening MG.
Study Rationale and Significance
Subcutaneous immunoglobulin (SCIG) is a novel form of immune therapy for neuromuscular diseases and may offer several advantages over intravenous immunoglobulin (IVIG).
Although no large-scale studies have been in conducted in MG patients, pilot studies with SCIG in multifocal motor neuropathy indicate that it may be a feasible, effective and safe alternative to IVIG in a condition, which like MG, shows excellent response to IVIG infusion.
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
- •Patients between18 to 80 years of age, diagnosed with MG (see below) who have worsening myasthenic symptoms - (defined as increasing diplopia, ptosis, dysarthria, dysphagia, difficulty chewing, or limb weakness severe enough to warrant immunoglobulin therapy.
- •MG diagnosis will be based upon the clinical evaluation by a neuromuscular expert and meeting any two of the following supportive criteria:
- •Abnormal Tensilon test
- •Abnormal repetitive nerve stimulation studies
- •Abnormal single fiber electromyography (EMG)
- •Increased serum acetylcholine receptor or anti-MuSK antibodies
- •Prior response to immunotherapy
排除标准
- •Respiratory distress requiring ICU admission or a vital capacity <1 L
- •Severe swallowing difficulties with a high risk of aspiration
- •Change in corticosteroid dosage in the 4 weeks prior to screening
- •Known immunoglobulin A (IgA) deficiency
- •Pregnant or breast feeding women
- •Active renal or hepatic insufficiency, clinically significant cardiac disease
- •Patients with worsening weakness associated with an infectious process
- •Previous lack of responsiveness to IVIG
- •History of previous MG crises
研究组 & 干预措施
SC Treatment Period
Participants will receive 2gm/kg of Human normal immunoglobulin G (IgG) infused over 4 weeks in a dose escalating manner as follows:
-
1st week: 2-3 SCIG infusions of 10ml per site at four sites (total dose 16 to 24g)*
-
2nd week: 2-3 SCIG infusions of 15ml per site at four sites (total dose 24 to 36g)*
-
3rd week: 2-4 SCIG infusions of 20ml per site at four sites (total dose 32 to 64g)*
-
4th week: 2-4 SCIG infusions of 25ml per site at four sites (total dose 40 to 80g)*
-
Doses indicated are study recommended. Doses may be adjusted depending on tolerance and total dose required by the patient.
干预措施: Human normal immunoglobulin G (IgG) (Drug)
结局指标
主要结局
Change in Quantitative Myasthenia Gravis Score (QMGS) from baseline to day 42 after treatment.
时间窗: Baseline - Day 42
QMGS is a validated clinical measure of myasthenia gravis ranging from 0 points (no myasthenic weakness) to a maximum of 39 points, with a defined change of 3.4 units required for clinical significance.
次要结局
- Quality of life will be assessed through the Quality of Life (QOL) score, a qualitative questionnaire.(Baseline - Day 42)
- Change in Manual Muscle Testing (MMT) score from baseline to day 42 after treatment.(Baseline - Day 42)
- Adverse events related to SCIG infusions will be recorded if/when they occur.(Baseline - Day 42)
- Patient satisfaction with the treatment modality will be assessed using a questionnaire.(Day 42)
- Serious Adverse Events related to SCIG infusions will be recorded if/when they occur.(Baseline - Day 42)
- Proportion of participants successfully trained will be recorded indicating feasibility based on patient compliance.(Baseline - Week 2)
- Proportion of participants completing will be recorded indicating feasibility based on patient compliance.(Baseline - Day 42)
- Myasthenia Gravis (MG) Composite scores will be used to evaluate disease severity through a number of functional assessments, including muscle strength and ability to complete activities of daily living.(Baseline - Day 42)