臨床研究等提出・公開システム

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

Multicenter trial of SPP-004 in mitochondrial diseases

Akira Otake

Saitama Medical University Hospital

38 Morohongo, Moroyama-machi, Iruma-gun, Saitama 350-0495, Japan

akira_oh@saitama-med.ac.jp

Kazuo Nakamura

CTD Inc.

3-3-2Tsukiji,Chuo-ku,Tokyo,140-0045,Japan

+81-3-6228-4922

mrcd@c-ctd.co.jp

COMPLETED

Dec. 10, 2014

Interventional

Multicenter, randomized, double-blind, placebo-controlled, clinical trial

double blind

Yes

2

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

Both

Mitochondrial disease, mainly to cranial nerve symptoms

Intervention type:DRUG Name of intervention:SPP-004 Dose form / Japanese Medical Device Nomenclature:CAPSULE Route of administration / Site of application:ORAL Dose per administration: 5-ALA HCl 25 and SFC 39.2 mg Dosing frequency / Frequency of use:BID Planned duration of intervention:24 weeks 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. detailes of teratment arms: Comparative intervention name:Placebo Dose form / Japanese Medical Device Nomenclature:CAPSULE Route of administration / Site of application:ORAL Dose per administration: 5-ALA HCl placebo and SFC 39.2 mg Dosing frequency / Frequency of use:BID Planned duration of intervention:12 weeks 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.

The Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Section I-III

Life prognosis
NPMDS Section I-IV
FGF21
Weight
Height
Head circumference
Respiratory chain complexes enzyme activity in dermal fibroblast (if informed consent is obtained)

Sept. 30, 2014