JPRN-jRCT2091220190已完成2 期
Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy - SMART01
Institute of Medical Genetics, Tokyo Women's Medical University0 个研究点目标入组 13 人开始时间: 2014年8月1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
- 13
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
- ot applicable 至 < 8age old（—）
- 性别
- All
入选标准
- •(1) Patients with SMN gene mutation proven by gene testing
- •(2) patients of SMA types 1, 2 and 3.
- •(3) Patients under age 8 when agreement was obtained. (both sexes accepted)
- •(4) Patients who can visit the hospital as scheduled in clinical trials.
- •(5)Patients with a representative (guardian) under conditions below
- •1)A fixed representative (guardian) can comprehend the status of the patient and observe the patient's daily activities as required in the clinical trial.
- •2)A fixed representative (guardian) can attend the hospital visits throughout the clinical trial period.
- •3)Management of medicine is definitely possible.
排除标准
- •(1)Patients who have had an operation for spine scoliosis.
- •(2)Patients on mechanical ventilator with tracheotomy or who are judged to require mechanical ventilation with tracheotomy by the investigator (or sub-investigator).
- •(3)Patients who have received sodium valproate before the agreement was obtained.
- •(4)Patient with severe liver dysfunction, renal dysfunction, and circulatory diseases. (judged referring to grade 3 of Severity Criteria for Drug Adverse Reaction Yaku-Ann No 80, issued by the chief of Registration Division the Pharmaceutical Affairs Bureau, Ministry of Welfare)
- •(5)Patients given Riluzole, creatine, butyric acid compound, and steroid hormone within 8 weeks before the agreement was obtained.
- •(6)Patients with epilepsy
- •(7)Patients with malignant tumors
- •(8)Patients with severe medical history of drug allergy such as anaphylactic shock, or serious side effect of drug.
- •(9)Patients participating in other clinical trials and administered an investigational new drug within 12 weeks before obtaining the agreement for this clinical trial.
- •(10) Patients scheduled to take a carbapenem antibiotic within the period from agreement to the end of observation and investigation, which will take 14 weeks.
- •(11) Patients with urea cycle abnormalities.
- •(12) Other patients judged to be unsuitable to join this clinical trial by the investigator (or sub-investigator).
研究者
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