NIPH Clinical Trials Search

JAPANESE
国立保健医療科学院
JMACCT ID: JMA-IIA00358

Registered date:22/06/2018

Multicenter trial of SPP-004 in mitochondrial diseases (confirmatory trial)

Basic Information

Recruitment status Completed
Health condition(s) or Problem(s) studiedMitochondrial disease, involved mainly central nervous system
Date of first enrollment30/06/2018
Target sample size40
Countries of recruitmentJapan
Study typeINTERVENTIONAL
Intervention(s)Intervention type:DRUG. Intervention1:SPP-004, Dose form:CAPSULE, Route of administration:ORAL, intended dose regimen:Open period A capsule of each investigational drug (5-ALA HCl capsule (25 mg) and SFC capsule (39.22 mg)) is administered orally or by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration. Double-blind period. Same dose in open period is administered orally or by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration. Through the study treatment (open period and double-blind period), doses are based on the body weight at observation time during the treatment period.. Control intervention1:Placebo, Dose form:CAPSULE, Route of administration:ORAL, Intended dose regimen:Open period A capsule of each investigational drug (5-ALA HCl capsule (25 mg) and SFC capsule (39.22 mg)) is administered orally or by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration. Double-blind period. Same dose in open period is administered orally or by a feeding tube. If capsules cannot be swollen, the content should be dissolved at the time of administration. Through the study treatment (open period and double-blind period), doses are based on the body weight at observation time during the treatment period..

Outcome(s)

Primary OutcomeThe proportion of subject meeting the criteria of "insufficient" at the week of 48 in the double-blind period.. Timepoint:Double-blind period 48w.
Secondary Outcome1.The proportion of subject meeting the criteria of "insufficient" at the week of 24 and 36 in the double-blind period. 2.Period (days) from starting "double-blind period" to the timing when investigator determined as "insufficient" 3.The Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Section I~III 4.NPMDS Section I,II,III, each 5.NPMDS Section I~IV, tatally 6.Blood FGF21 7.Body Weight 8.Body Height 9.Head circumference 10.Blood GDF-15 11.Respiratory chain complex enzymes activity and oxygen consumption in skin fibroblasts (if informed consent is obtained) 12.General improvement 13.MRI of Brain 14.Life prognosis 15.Period of ventilator weaning 16.Number of "stroke-like episodes" (applicable for the subject who had a history of stroke-like episodes before participation of the clinical trial). Timepoint: 1, Double-blind period 24w and 36w 2, Double-blind period 48w 3-10, Every 12ws 11, Every 24ws 12, Every 12ws 13, Double-blind period 24w and 48w 14-16, Every 12ws.

Key inclusion & exclusion criteria

Age minimum>=3 MONTHS
Age maximumNo Limit
GenderBOTH
Include criteriaJapanese patients who meet all the following criteria are included in this clinical trial. (1)Subject who meets all three clinical criteria of suspicious for Leigh syndrome as follows: 1.have progressive central nervous system symptoms 2.have symptoms of brainstem and/or basal ganglia lesions 3.Have symmetrical necrotizing lesions in brainstem and/or basal ganglia confirmed by imaging data or pathological examination*. *bilateral and symmetric brainstem and/or basal ganglia lesions are observed,as low-density area by brain CT, or as high-intensity area by T2-weighted or FLAIR image in brain MRI, or multifocal and symmetrical lesions are confirmed at basal ganglia, thalamus, brainstem, dentate nucleus and optic nerves by pathological examination. (2)Patients aged>=3months at the timing of IC acquisition (3) Patients taking a food containing 5-ALA at the timing of IC acquisition and medically allowed to stop taking the food for the 28 consecutive days before starting open period (4) Patient's legally authorized representative or subject must provide written informed consent** **for a patient who have a competent to consent to IC must provide assent, patient needs to provide a written IC even in the case which patient's legally authorized representative provides written informed consent
Exclude criteriaPatients who meet any of the following criteria are excluded from this trial. (1)Patients diagnosed as Vitamin B1 deficiency or organic acidemias(ex: Methylmalonic acidemia, Glutaric acidemia type1) (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 with a pregnant or breast-feeding woman, or woman suspected of being pregnant (8)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 +81-49-276-1218
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 +81-49-276-1218
E-mail
Affiliation Saitama Medical University Hospital