Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 23
- 试验地点
- 5
- 主要终点
- Proportion of Patients Whose Quantitative Myasthenia Gravis Scores Are Increased by no More Than 3 Points at the End of the SCIg Treatment Phase
研究概览
简要总结
The purpose of this study is to determine whether Hizentra is a safe and effective treatment for people with myasthenia gravis (MG).
详细描述
Myasthenia gravis (MG) is a rare autoimmune disorder which causes the muscles to become weak because the immune system attacks the connection between the nerves and the muscles.
Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). An immunoglobin is a blood protein. Hizentra is being studied for the treatment of patients with MG. Hizentra is administered by an injection into the skin through a portable infusion pump, which may be easier for patients to administer than the current treatments.
Participants will be asked to complete 9 clinic visits and 3 telephone calls. It could take up to 30 weeks to complete all study visits.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients 18 and older
- •Patients must have prior or current documentation of MGFA MG grades 2, 3, or 4 generalized MG, according to the MGFA classification system.48 These grades correspond to mild (2), moderate (3), and severe (4)
- •Elevated AChR or MuSK Ab. These tests will have been performed at some time prior to entry into the study. Double seronegative MG patients with prior documentation of an abnormal decrement (>10%) on slow repetitive nerve stimulation or an abnormal single fiber EMG will also be allowed to participate
- •Patient's signs and symptoms should not be better explained by another disease process.
- •IVIg maintenance dose of 0.2 to 2 gm/kg/4 weeks or equivalent dose administered Q 2-4 weeks±3days
- •Stable IVIg for at least 3 cycles (definition of stability: no change in prescribed dosage or frequency by the treating physician)
- •Patient must be receiving no more than 200g/4weeks of IVIg.
- •Patients must be willing to complete the study and return for follow-up visits.
- •Patients must be willing to give written informed consent before participating in this study. A copy of the signed consent must be kept in the patient's medical record.
- •Patients can be on the following drugs as long as there has been no dose change for 60 days: azathioprine, cyclosporine, cyclophosphamide, mycophenolate mofetil, tacrolimus, methotrexate or other immunosuppressive drugs.
- •Patients can be on prednisone as long as there has been no dose change for 30 days.
排除标准
- •MGFA grade V within 6 months of screening.
- •A history of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue.
- •Other major chronic or debilitating illnesses within six months prior to study entry.
- •Female patients who are premenopausal and are: (a) pregnant on the basis of a serum pregnancy test, (b) breast-feeding, or (c) not using an effective method of double barrier (1 hormonal plus 1 barrier method or 2 simultaneous barrier methods) birth control (birth control pills, male condom, female condom, intrauterine device, Norplant, tubal ligation, or other sterilization procedures).
- •Altered levels of consciousness, dementia, or abnormal mental status.
- •Thymectomy in the previous three months.
- •Evidence of renal insufficiency (Cr>1.5 x elevated) or liver disease (transaminases > 2.5 x elevation) at screening.
- •Skin disease that would interfere with assessment of injection site reaction
- •History of severe reactions to IVIg or SCIg.
- •Participation in a research study within the last 3 months
- •Treatment with rituximab or other biologics within 12 months of study entry
- •Inability to provide informed consent.
- •History of thrombotic episodes within the last year prior to enrollment
- •Known allergic or other severe reactions to blood products including intolerability to previous normal human immunoglobulin for intravenous administration (IVIG) and/or subcutaneous immunoglobulin (SCIG), such as history of clinically relevant hemolysis after IVIG infusion, aseptic meningitis, recurrent severe headache, hypersensitivity, severe generalized or severe local skin reaction.
- •History of IgA deficiency or evidence of IgA deficiency at screening.
研究组 & 干预措施
HIZENTRA ®
Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). Participants will receive Hizentra in a minimum of one infusion per week and a maximum of 4 infusions per week. Dose and rate depend on the visit and how each participant tolerates the drug. Max cc per site is 50 cc per site per hour.
干预措施: HIZENTRA ® (Drug)
结局指标
主要结局
Proportion of Patients Whose Quantitative Myasthenia Gravis Scores Are Increased by no More Than 3 Points at the End of the SCIg Treatment Phase
时间窗: Change from Baseline to Week 12
The QMG is a 13 item ordinal scale which measures ocular, bulbar, extremity fatigue and strength, along with respiratory function. The scale is from 0 - 3 for each item, with 0 meaning normal and 3 is severe. Total score can range from 0 to 39. Change in MG severity will be measured using the Quantitative Myasthenia Gravis (QMG) Score for Disease severity. The QMG is a validated clinical composite scale. As mentioned in the protocol, our hypotheses are: H0: Proportion of patients whose QMG scores are increased by more than 3 points at the end of the SCIg treatment phase ≤ 0.65 HA: Proportion of patients whose QMG scores are increased by no more than 3 points at the end of the SCIg treatment phase \> 0.65 Thus, analysis of the primary outcome is done as a one-sample Z test of proportions. That is, the QMG is a continuous outcome, but analyses results are reported as proportions.
次要结局
- Myasthenia Gravis-specific Activities of Daily Living Scale (MG-ADL) Scores(Change from Baseline to Week 12)
- Myasthenia Gravis Composite (MGC) Score(Change from Baseline to Week 12)
- Treatment Satisfaction Questionnaire for Medication (TSQM) - Effectiveness Score(Change from Baseline to Week 12)
- Treatment Satisfaction Questionnaire for Medication (TSQM) - Satisfaction Score(Change from Baseline to Week 12)
- Myasthenia Gravis Quality of Life (MG QOL-15) Scores(Change from Baseline to Week 12)
- Treatment Satisfaction Questionnaire for Medication (TSQM) - Convenience Score(Change from Baseline to Week 12)
- Immunoglobulin G (IgG) Antibody Levels(Change from "Week -10 to Week 0" versus "Week 1 to Week 12")
- Tolerabililty(12 weeks from start of SCIg)
研究者
Mazen Dimachkie, MD
Professor
University of Kansas Medical Center