NIPH Clinical Trials Search

JAPANESE
国立保健医療科学院
JMACCT ID: JMA-IIA00214

Registered date:26/05/2015

Long-term, 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 enrollment02/06/2015
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: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..

Outcome(s)

Primary OutcomeThe Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Section I~III. Timepoint:Screening,Treatment period every 12 weeks, 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 every 12 weeks, discontinuation. Respiratory chain complexes enzyme activity in dermal fibroblasts, Screening, after 48, 96, 144, and 180 weeks, and at discontinuation..

Key inclusion & exclusion criteria

Age minimum>=9 MONTHS
Age maximum<30 MONTHS
GenderBOTH
Include criteriaJapanese 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)
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, 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

Related Information

Contact

public contact
Name Akira Otake
Address Department of Pediatrics,38 Morohongo, Moroyama-machi, Iruma-gun, Saitama 350-0495, Japan
Telephone +81-49-276-1111
E-mail akira_oh@saitama-med.ac.jp
Affiliation Saitama Medical University Hospital
scientific contact
Name Akira Otake
Address Department of Pediatrics,38 Morohongo, Moroyama-machi, Iruma-gun, Saitama 350-0495, Japan
Telephone +81-49-276-1111
E-mail
Affiliation Saitama Medical University Hospital