JPRN-jRCT2031210252招募中1 期
A multicenter phase 1 study of NS-035 in patients with Fukuyama-type congenital muscular dystrophy
Toda Tatsushi0 个研究点目标入组 12 人开始时间: 2021年8月18日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 5age old 至 <= 10age old（—）
- 性别
- All
入选标准
- •1) Patients with homozygous or complex heterozygous mutations in the fukutin gene and a definitive diagnosis of FCMD
- •2) Patients who do not have DNA polymorphism that may compromise double helix formation with NS-035, as judged by DNA sequencing of retrotransposon insertion mutations in the 3'untranslated region of the fukutin gene
- •3) Patients aged 5 to 10 at the time of consent
- •4) Patients whose legal guardian capable of providing informed consent has provided written informed consent upon thorough understanding of the study procedure. Efforts should be made so that the subjects themselves give voluntary assent after having been provided with an explanation according to their ability to understand.
- •5) Patients who are expected to survive until the end of this trial
- •6) Patients who have intact muscles allowing appropriate evaluation of the effect of the study drug (neither loss nor severe atrophy of the deltoid or the biceps brachii muscle)
- •7) Patients who can maintain a sitting position without assistance (FCMD motor function level (Ueda classification) is >=2)
- •8) Patients with QTc (based on Fridericia's correction) <450 msec on 12-lead ECG performed during the pre-observation period (patients with QTc <480 msec if bundle branch block is observed)
排除标准
- •1) Patients with percutaneous arterial oxygen saturation (SpO2) <95%
- •2) Patients with Left ventricular ejection fraction (LVEF) <50% or fractional shortening (FS) <25% based on echocardiogram performed during the pre-observation period
- •3) Patients with a history of epilepsy who have had status epilepticus within the past year since the consent was obtained.
- •4) Patients who are continuously using a ventilator (NPPV can be used while sleeping)
- •5) Patients whose serum cystatin C exceeds the upper limit of the institutional reference value in the tests performed during the pre-observation period and is judged to be clinically variable
- •6) Patients undergoing gastric tube feeding
- •7) Patients who used systemic corticosteroids within 3 months before the start of study drug administration
- •8) Patients who underwent surgery within 3 months before the start of administration of the investigational drug and patients who are scheduled to undergo surgery after provisional registration until the end of the post-observation period
- •9) Patients with active or uncontrollable infections, cardiomyopathy, liver disease and kidney disease
- •10) Patients with acute intracranial hematoma on MRI examination performed during the pre-observation period
- •11) Patients with dehydration or electrolyte imbalance in the pre-observation period
- •12) Patients with renal dysfunction such as urinary retention or diabetic nephropathy
- •13) Patients with dysphagia
- •14) Patients who are positive for HBsAg, HCV antibody, HIV antibody or syphilis test
- •15) Patients with immunodeficiency or autoimmune disease
- •16) Patients who received other investigational drugs or study drugs in clinical studies within 3 months before the start of administration of the study drug
- •17) Patients with a history of severe drug hypersensitivity
- •18) Pregnant patients or potentially pregnant patients who do not agree to contraception during the trial period (including menarche during the trial period)
- •19) Patients who are judged by the investigator (or subinvestigator) to be inappropriate for this clinical trial for any reason
研究者
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