Multicenter trial of SPP-004 in mitochondrial diseases (confirmatory trial) (MITO Study)

Saitama Medical University Hospital0 个研究点开始时间: 待定最近更新: 2021年7月11日

试验速览

暂无简介。

•Japanese patients who meet all the following criteria are included in this clinical trial.
•(1)Subject who meets all three clinical criteria of suspicious for Leigh syndrome as follows:
•1.have progressive central nervous system symptoms
•2.have symptoms of brainstem and/or basal ganglia lesions
•3.Have symmetrical necrotizing lesions in brainstem and/or basal ganglia confirmed by imaging data or pathological examination*.
•bilateral and symmetric brainstem and/or basal ganglia lesions are observed,as low-density area by brain CT, or as high-intensity area by T2-weighted or FLAIR image in brain MRI, or multifocal and symmetrical lesions are confirmed at basal ganglia, thalamus, brainstem, dentate nucleus and optic nerves by pathological examination.
•(2)Patients aged≧3months at the timing of IC acquisition
•(3) Patients taking a food containing 5-ALA at the timing of IC acquisition and medically allowed to stop taking the food for the 28 consecutive days before starting open period
•(4) Patient's legally authorized representative or subject must provide written informed consent**
•*for a patient who have a competent to consent to IC must provide assent, patient needs to provide a written IC even in the case which patient's legally authorized representative provides written informed consent

•Patients who meet any of the following criteria are excluded from this trial.
•(1)Patients diagnosed as Vitamin B1 deficiency or organic acidemias(ex: Methylmalonic acidemia, Glutaric acidemia type1)
•(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 with a pregnant or breast-feeding woman, or woman suspected of being pregnant
•(8)Patients whom the investigator and sub-investigator considered inappropriate to participating this trial

proportion of subject meeting the criteria of "insufficient"

时间窗: week of 48 in the double-blind period

The proportion of subject meeting the criteria of "insufficient"

The proportion of subject meeting the criteria of "insufficient" at the week of 24 and 36 in the double-blind period(week of 24 and 36 in the double-blind period)
MRI of Brain
Period (days) from starting "double-blind period" to the timing when investigator determined as "insufficient"
The Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Section I~III
NPMDS Section I,II,III, each
NPMDS Section I~IV, tatally
Blood FGF21
Body Weight
Body Height
Head circumference
Blood GDF-15
Respiratory chain complex enzymes activity and oxygen consumption in skin fibroblasts (if informed consent is obtained)
Period of ventilator weaning
General improvement
Life prognosis
Number of "stroke-like episodes" (applicable for the subject who had a history of stroke-like episodes before participation of the clinical trial)

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