臨床研究等提出・公開システム

Long-term, multicenter trial of SPP-004 in mitochondrial diseases. (MRCD Long- term Study)

Long-term, multicenter trial of SPP-004 in mitochondrial diseases.

9

Akira Otake

Saitama Medical University Hospital

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

akira_oh@saitama-med.ac.jp

Akira Otake

Saitama Medical University Hospital

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

+81-49-276-1111

akira_oh@saitama-med.ac.jp

COMPLETED

June. 02, 2015

Interventional

Unblinded, uncontrolled, long-term, multicenter trial

open(masking not used)

No

2

Japanese patients who meet all of the following criteria are included in this clinical trial.
1)Patients participated in, and completed, an exploratory study of SPP-004 in mitochondrial diseases (study protocol no.: SPED-ALA-001)
2)Patients with written informed consent from their legal representatives (persons in parental authority or guardians)

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, except those who participated in the exploratory study of SPP-004 in mitochondrial diseases (study protocol no.SPED-ALA-001)
7)Patients whom the investigator and sub-investigator 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 25-75 / SFC 39.22-117.66 mg Dosing frequency / Frequency of use:BID Investigational drug doses are based on the body weight at each observation time during the treatment period. Planned duration of intervention:216 weeks Intended dose regimen:5-ALA HCl and SFC capsules are administered orally or by a feeding tube in accordance with doses and frequency of administration based on body weight at each observation time during the period of treatment. If capsules cannot be swollen, the content should be dissolved at the time of administration. detailes of teratment arms: Comparative intervention name: Dose form / Japanese Medical Device Nomenclature: Route of administration / Site of application: Dose per administration: Dosing frequency / Frequency of use: Planned duration of intervention: Intended dose regimen:

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)

April. 28, 2015