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JAPANESE
国立保健医療科学院
JMACCT ID: JMA-IIA00200

Registered date:09/12/2014

Multicenter trial of SPP-004 in mitochondrial diseases

Basic Information

Recruitment status Completed
Health condition(s) or Problem(s) studiedMitochondrial disease, mainly to cranial nerve symptoms
Date of first enrollment10/12/2014
Target sample size10
Countries of recruitmentJapan
Study typeINTERVENTIONAL
Intervention(s)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. .

Outcome(s)

Primary OutcomeThe Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Section I-III. Timepoint:Screening, Treatment period 8,12 and 20,24 weeks or discontinuation.
Secondary OutcomeLife 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.

Key inclusion & exclusion criteria

Age minimum>=3 YEARS
Age maximum<2 YEARS
GenderBOTH
Include criteriaJapanese 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
Exclude criteriaPatients 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

Related Information

Contact

public contact
Name Kazuo Nakamura
Address Pharmaceutical Promotion Department,3-3-2Tsukiji,Chuo-ku,Tokyo,140-0045,Japan
Telephone +81-3-6228-4922
E-mail mrcd@c-ctd.co.jp
Affiliation CTD Inc.
scientific contact
Name Akira Otake
Address Department of Pediatrics,38 Morohongo, Moroyama-machi, Iruma-gun, Saitama 350-0495, Japan
Telephone +81-49-276-1218
E-mail
Affiliation Saitama Medical University Hospital