Multicenter trial of SPP-004 in mitochondrial diseases

INTERVENTION: Intervention type:DRUG. Intervention1:SPP‐004, Dose form:CAPSULE, Route of administration:ORAL, intended dose regimen:A capsule of each investigational drug [5‐ALA HCl capsule (25 mg) and SFC capsule (39.22 mg)] is administered twice a day orally or by a feeding tube . After Week 13, a capsule of each investigational drug (5‐ALA HCl capsule and SFC capsule) is administered twice a day orally and by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration.. Control intervention1:Placebo, Dose form:CAPSULE, Route of administration:ORAL, Intended dose regimen:A capsule of each investigational drug [5‐ALA HCl placebo capsule (25 mg) and SFC capsule (39.22 mg)] is administered twice a day orally or by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration. . CONDITION: Mitochondrial disease, mainly to cranial nerve symptoms PRIMARY OUTCOME: The Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Section I‐III. Timepoint:Screening, Treatment period 8,12 and 20,24 weeks or discontinuation. SECONDARY OUTCOME: Life prognosis; NPMDS Section I‐IV; FGF21; Weight; Height; Head circumference; Respiratory chain complexes enzyme activity in dermal fibroblast (if informed consent is obtained). Timepoint: Screening, Treatment period 8,12 and 20,24 weeks or discontinuation. INCLUSION CRITERIA: 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