NCT04965987撤回1 期
A Phase I, Double-Blind, Pilot Study of Oxaloacetate in Myasthenia Gravis
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 撤回
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Safety (Frequency and Severity of Adverse Events)
研究概览
简要总结
This is a phase 1 clinical trial studying whether or not oxaloacetate has a positive effect on patients with Myasthenia Gravis. Patients will be assigned to one of three cohorts which will determine the dose of oxaloacetate they will be given. Subjects will take the study drug for 4 weeks and be on placebo for 4 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, 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
- •Patients 18 and older.
- •Patients must have prior or current documentation of MGFA MG grades 2, 3, or 4A generalized MG, according to the MGFA classification system. These grades correspond to mild (2), moderate (3), and severe (4).
- •Patient's signs and symptoms should not be better explained by another disease process.
- •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, IVIg or other immunosuppressive drugs.
- •Patients can be on prednisone as long as there has been no dose change for 30 days.
- •No planned changes in MG medications during the study
排除标准
- •MGFA grade V within 6 months of screening.
- •A history of chronic degenerative, severe 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).
- •Thymectomy in the previous three months.
- •History of severe reactions to OAA
- •Participation in a research study within the last 3 months
研究组 & 干预措施
Oxaloacetate
Active Comparator
Oxaloacetate (OAA) is a four-carbon molecule involved in many metabolic pathways, including gluconeogenesis, citric acid cycle, glyoxylate cycle, urea cycle, and amino acid metabolism. In the glyoxylate and citric acid cycles, oxaloacetate is formed as the result of the catalysis by malate dehydrogenase.
Subjects will take 1000mg OAA BID for 8 weeks.
干预措施: Oxaloacetate (Drug)
Placebo
Placebo Comparator
Subjects will take 1000mg Placebo BID for 8 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Safety (Frequency and Severity of Adverse Events)
时间窗: At weeks 2, 4, 6, 8, 10, and 12
The primary endpoint of safety will be reported as counts of subjects experiencing adverse events events (including abnormal laboratory results and vital signs). Measures of safety will be reported for the last visit observed for each participant.
次要结局
- Myasthenia Gravis-specific Activities of Daily Living Scale (MG-ADL) Score(Change from Week 4 to Week 8)
- Quantitative Myasthenia Gravis (QMG) Score(Change from Week 4 to Week 8)
研究者
研究点 (1)
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