2023-507539-40-00招募中2 期
A Phase 2b, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of 3 Dose Levels of NMD670 over 21 Days in Adult Patients with AChR/MuSK-Ab+ Myasthenia Gravis
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 42
- 试验地点
- 23
- 主要终点
- Change from baseline to day 21 in quantitative myasthenia gravis (QMG) total score for NMD670 vs placebo
研究概览
简要总结
To assess dose response of NMD670 on clinical efficacy as rated by clinicians
研究设计
- 分配方式
- Randomized
- 主要目的
- Multicenter, randomised, double-blind, placebo-controlled
- 盲法
- Double (Monitor, Subject, Analyst, Investigator, Carer)
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Participant must be a male or female being ≥18 years, at the time of signing the informed consent.
- •Diagnosis of MG, MGFA class II, III or IV
- •Documented positive AChR or MuSK antibody test.
- •Participant must be able to swallow tablets
- •Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg
- •Female participants who are WOCBP and male participants with partners who are WOCBP must agree to use a highly effective contraception method during the study. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- •Participant is capable of and has given signed informed consent.
排除标准
- •Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient’s full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study.
- •Active or untreated thymoma, a history of thymic carcinoma or thymic malignancy (unless deemed cured by adequate treatment with no evidence of recurrence for 5 years or more before screening), a history of thymectomy in the 6 months before screening.
- •Participant with a clinical diagnosis of gout, or with serum uric acid >upper limit of normal (ULN) at screening.
- •Participant with any of the following: a. Abnormal liver function test defined as total bilirubin >1.5× ULN (except for participants with known Gilbert’s syndrome). b. Abnormal liver transaminase levels at baseline and confirmed current or chronic history of liver disease including (but is not limited to) hepatitis virus infections, drug- or alcohol-related liver disease, non alcoholic steatohepatitis, autoimmune hepatitis, hemochromatosis, Wilson’s disease, α 1 antitrypsin deficiency, primary biliary cholangitis, primary sclerosing cholangitis, or any other liver disease considered clinically significant by the Investigator. c. Known hepatic or biliary abnormalities (with the exception of Gilbert’s syndrome or asymptomatic gallstones). d. Any renal impairment or kidney abnormalities considered clinically significant by the Investigator at screening.
- •Participant with laboratory test abnormalities at screening considered clinically significant by the Investigator.
- •Participants received treatment with IMP within 30 days (or 5 half-lives of the medication, whichever is longer) prior to day
- •Participants with history of poor compliance with relevant MG therapy.
- •Female patients who plan to become pregnant during the study or are currently pregnant or breastfeeding
结局指标
主要结局
Change from baseline to day 21 in quantitative myasthenia gravis (QMG) total score for NMD670 vs placebo
Change from baseline to day 21 in quantitative myasthenia gravis (QMG) total score for NMD670 vs placebo
次要结局
未报告次要终点
研究者
Vera Kiyasova
Scientific
NMD Pharma A/S
研究点 (23)
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