JPRN-jRCT1091220134已完成2 期
Phase II study of nonsense readthrough compound NPC-14 (Arbekacin sulfate) to explore safety, tolerability, and efficacy in Duchenne muscular dystrophy patients (NORTH POLE DMD study) - NORTH POLE DMD study
Yasuhiro Takeshima, MD, PhDHyogo College of Medicine Hospital, department of pediatrics0 个研究点目标入组 21 人开始时间: 2013年8月5日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 21
研究概览
简要总结
Good safety and tolerability of NPC-14 were shown in ambulant DMD patients by adjustment of dosage to 40 micro g/mL of Cmax. Change of dystrophin expression rate, which is primary outcome of NPC-14 effect, is difficult to be judged because of lower expression than sensitivity of the measurement. Assessment of dystrophin expression by more sensitive assay system is necessary.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 4age old 至 ot applicable（—）
- 性别
- Male
入选标准
- •(1)Diagnosis of DMD resulting from a nonsense mutation by whole genome sequencing of the dystrophin gene
- •(2)To have intact right or left biceps muscles, or an alternative muscle group that is able to be underwent for appropriate evaluation of efficacy
- •(3)To meet the following criteria at screening (baseline visit), within 30 days prior to the first dose of study drug
- •-Ambulant and able to walk at least 75 meters during the 6MWT
- •-Able to comply with and complete all protocol requirements, and judged by the investigator to be appropriate to participate in the study from the screening results
- •(4)Aged at least 4 years at the time of giving informed consent
- •(6)Able to be hospitalized for the study requirement
- •(7)Signed Informed consent by parents/legal guardian and/or signed assent by the subject (age of assent to be determined by IRB)
排除标准
- •(1) Prior exposure to investigational medicines that have a potential of restoring dystrophin or other functional protein (readthrough, exon skipping, utrophin upregulation therapy etc.)
- •(2) Known mutation at nucleotide 1555 in 12S rRNA gene of mitochondrial DNA, and/or personally or families have been treated or have a history of eight cranial nerve disorder (hearing loss,vertigo,tinnitus etc.)as a result of aminoglycoside use
- •(3) Inability to hear within the range of 0 to 25 dB by pure tone audiometry, abnormalities on auditory brainstem response audiometry, and/or loss of frequency by distortion product oto acoustic emissions at screening
- •(4) Poor oral intake or enable to oral intake, and/or bad general status
- •(5) Known allergies to NPC-14, other aminoglycosides, and/or bacitracin
- •(6) Presence of anti-dystophin antibody at the baseline assessments
- •(7) Cys-C >=1.2 mg/L and/or creatinine concentration >1.5 times the upper limit of age corrected normal range
- •(8) Left ventricular ejection fraction (EF) <40% or left ventricular fractional shortening (FS) <25%, and/or >=480 msec QTc (corrected QT interval by Fridericia's method)
- •(9) Need of mechanical ventilation
- •(10) Forced vital capacity (FVC) <50% predicted
- •(11) Clinically significant concomitant diseases (hematology, psychoneurotic, hepatic, pulmonary, endocrine, immune, renal, and gastroenterological diseases), and/or cancer
- •(12) Impairment of intellectual functions, and/or expressive language ability which might interfere with study assessments
- •(13) Treatment with other systemic gminoglycoside within 6 months prior to the first administration of study drug
- •(14) Initiation of systemic glucocorticosteroids treatment, and/or start exercise cure, physical therapy, or occupational therapy which might interfere with study assessments. Changing of dose and schedule of systemic glucocorticosteroids within 6 months prior to the first administration of study drug
- •(15) History of any surgical procedure within months prior to the first administration of study drug or have a plan during study
- •(16) History of sever allergy from food and medicine like an anaphylaxis shock or generalized rash
- •(17) Participation in any other clinical trial and intake of any investigational drug within 6month of study entry
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