Pharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Change in serum concentration of N-acetylneuraminic acid
研究概览
简要总结
The aim of this study is to investigate pharmacokinetics and safety of N-acetylneuraminic acid in patients with Distal myopathy with rimmed vacuoles (DMRV) - hereditary inclusion body myopathy (hIBM). Dosages are 800mg single and three times a day and 800mg 3 times a day for 5 consecutive days. N-acetylneuraminic acid and N-glycolylneuraminic acid in serum and urine are measured before and after oral administration of N-acetylneuraminic acid.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 20 Years 至 40 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed mutations in GNE gene
- •No severe complications when informed consent is obtained
- •More than 40 kg in weight before administration
排除标准
- •Hepatic laboratory parameters (AST, ALT, γ-GTP) or Renal laboratory parameters (Cr, BUN) are greater than three times of upper limit of reference value
- •Presence or history of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, gastrointestinal, brain, psychiatric, neurologic disease
- •Intake of supplement contains sialic acid, St. John's wort or grapefruit within 7 days
- •Enrollment in another investigational study within 3 months
- •More than 400 mL blood donation within 3 months
- •Presence of alcohol or drug dependency
- •Women who are pregnant , breast feeding or possible to be pregnant.
- •Patients whom the investigator judges not to be appropriate for the subject
研究组 & 干预措施
NPC-09
Period 1: NPC-09 800mg single oral dosing NPC-09 800mg three times oral dosing a day Period 2: NPC-09 800mg three times oral dosing a day for 5 consecutive days
干预措施: NPC-09 (Drug)
结局指标
主要结局
Change in serum concentration of N-acetylneuraminic acid
时间窗: 24 hours before dosing and dosing days
Patients with DMRV(hIBM) show low serum N-acetylneuraminic acid level.
Change in pharmacokinetic parameters of N-acetylneuraminic acid: AUCt
时间窗: 24 hours before dosing and dosing days
Change in amount of urinary excretion of N-acetylneuraminic acid
时间窗: 24 hours before dosing and dosing days
Safety(adverse events)
时间窗: Up to 5-7 days after dosing
次要结局
- Change in serum concentration of N-glycolylneuraminic acid(24 hours before dosing and dosing days)
- Change in pharmacokinetic parameters of N-glycolylneuraminic acid: AUCt(24 hours before dosing and dosing days)
- Change in amount of urinary excretion of N-glycolylneuraminic acid(24 hours before dosing and dosing days)