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

NMD Pharma A/S72 个研究点分布在 10 个国家目标入组 84 人2024年5月16日

适应症Myasthenia Gravis Myasthenia Gravis, MuSK

干预措施Placebo NMD670

概览

阶段: 2 期
干预措施: Placebo
疾病 / 适应症: Myasthenia Gravis
发起方: NMD Pharma A/S
入组人数: 84
试验地点: 72
主要终点: Change from baseline to day 21 in QMG total score for NMD670 vs placebo
状态: 招募中
最后更新: 19天前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验

概览

简要总结

This Phase 2 proof-of-concept, dose range finding study aims to evaluate the safety and efficacy of 3 dose levels of NMD670 vs placebo in adult patients with MG with antibodies against AChR or MuSK, administered twice a day (BID) for 21 days.

注册库

clinicaltrials.gov

开始日期

2024年5月16日

结束日期

2026年5月1日

最后更新

19天前

研究类型

Interventional

研究设计

Parallel

性别

All

研究者

发起方

NMD Pharma A/S

责任方

Sponsor

入排标准

入选标准

•Participant must be a male or female being 18 or more, 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
•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
•Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study
•Participants that received treatment with an investigational medical product within 30 days or 5 half-lives of the medication, whichever is longer prior to Day 1
•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

研究组 & 干预措施

Placebo

干预措施: Placebo

NMD670 high dose

干预措施: NMD670

NMD670 low dose

干预措施: NMD670

NMD670 mid dose

干预措施: NMD670

结局指标

主要结局

Change from baseline to day 21 in QMG total score for NMD670 vs placebo

时间窗: Baseline to day 21

Scale goes from 0-39 and higher score indicates worse symptomatology

次要结局

Change from baseline to day 21 in MGC total score for NMD670 vs placebo(Baseline to day 21)
Change from baseline to day 21 in MG-QOL15r for NMD670 vs placebo(Baseline to day 21)
Incidence of treatment emergent adverse event(Over 21 days of dosing)
Incidence of serious treatment emergent adverse events(Over 21 days of dosing)
Incidence of clinically significant abnormalities on physical examinations(Over 21 days of dosing)
Change from baseline to day 21 in MG-ADL total score for NMD670 vs placebo(Baseline to day 21)
Change from baseline to day 21 in Neuro-QoL Fatigue Short Form(Baseline to day 21)
Incidence of clinically significant abnormalities on safety laboratory parameters(Over 21 days of dosing)
Incidence of clinically significant ECG abnormalities(Over 21 days of dosing)
Incidence of Suicidal Ideation or Suicidal Behavior(Over 21 days of dosing)
Incidence of clinically significant abnormalities on opthalmological examinations(From screening (day -28 to day -1) until follow up (day 28))
Incidence of clinically significant vital signs abnormalities(Over 21 days of dosing)