Multicenter trial of SPP-004 in mitochondrial diseases - MRCD Study

Saitama Medical University Hospital0 个研究点目标入组 10 人开始时间: 2014年12月9日最近更新: 2023年10月17日

适应症

试验速览

暂无简介。

•Japanese patients who meet all of the following criteria are included in this clinical trial.
•1)Patients who were confirmed to be respiratory chain enzyme deficiency or mitochondrial gene abnormality by clinical findings in patients with suspected mitochondrial disease*
•2)Patients aged ? 3 months and < 2 years at the acquisition of consent forms (premature infants are dealt as corrected age in month)
•3) Patients with blood lactate-pyruvate ratio ?15.0
•4) Patients with written informed consent from their legal representatives (persons in parental authority or guardians)
•*Patients with all of the followings 1 to 3, as well as one or more of the conditions in 4
•1. Progressive neurological disorders associated with delayed motor and mental development
•2. Symptoms or signs of disease in brain stem and/or cerebral basal ganglia
•3. Increased lactate levels in blood and/or cerebrospinal fluid
•4. Patients who meet one or more of the followings
•1. Patients who have characteristic findings in images. (bilateral and symmetrical lesion of the brain stem or basal ganglia)
•2. Patients with typical neuropathological changes (spongiform degeneration)
•3. Patients with a sibling with similar symptoms

•Patients who meet any of the following criteria are excluded from this trial.
•1) Patients with mitochondrial cardiomyopathy
•2) Patients with severe cardiac function or renal function disorders
•3) Patients complicated by sepsis
•4) Patients with a history of drug allergy
•5) Patients with a history of hypersensitivity to any ingredients of the investigational drugs
•6) Patients participated in another clinical study within 12 weeks prior to the informed consent.
•7) Patients whom the investigator and subinvestigator considered inappropriate to participating this trial

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