NIPH Clinical Trials Search

JAPANESE
国立保健医療科学院
JMACCT ID: JMA-IIA00406

Registered date:18/01/2019

Long-term, multicenter trial of SPP-004 in mitochondrial diseases(after confirmation trial)

Basic Information

Recruitment status Completed
Health condition(s) or Problem(s) studiedMitochondrial disease, involved mainly central nervous system
Date of first enrollment01/02/2019
Target sample size55
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 screening and each observation time during the period of treatment.If capsules cannot be swollen, the content should be dissolved at the time of administration.. Control intervention1:-, Intended dose regimen:-.

Outcome(s)

Primary OutcomeSafety. Timepoint:Treatment period,study discontinuation and follow up.
Secondary Outcome1.Change in the Newcastle pediatric mitochondrial disease scale (NPMDS) for both central nervous system and neuromuscular symptom at the last point of administration from baseline 2.The Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Section I~III 3.NPMDS Section I,II,III, each 4.NPMDS Section I~IV, totally 5.Blood FGF21 6.Body Weight 7.Body Height 8.Head circumference 9.Blood GDF-15 10.Respiratory chain complex enzymes activity and oxygen consumption in skin fibroblasts (if informed consent is obtained) 11.General improvement rate evaluated by Investigator 12.Brain MRI 13.Life prognosis 14.period of ventilator weaning 15.Number of "stroke-like episodes" (applicable for the subject who had a history of stroke-like episodes before participation of the clinical trial of SPED-ALA-003). Timepoint: 1~4.end of study or study discontinuation 5.every 24 weeks 6~8.every 12 weeks 9.every 24 weeks 10.every 48 weeks 11.every 12 weeks 12.every 48 weeks 13.end of study or study discontinuation 14~15.every 12 weeks .

Key inclusion & exclusion criteria

Age minimum>=9 MONTHS
Age maximumNo Limit
GenderBOTH
Include criteriaJapanese patients who meet all the following criteria (1or2) are included in this clinical trial. 1. Subject participating on-going clinical trial SPED-ALA-002 (long-term, multicenter trial of SPP-004 in mitochondrial diseases) (1) Subject willing to participate into SPED-ALA-004 study (2) Subject meeting all eligibility criteria assessed by the investigator (3) Subject's legally authorized representative must provide written informed consent 2. Subject participated clinical trial of SPED-ALA-003 (confirmatory, multicenter trial of SPP-004 in mitochondrial diseases) (1) Subject participated clinical trial of SPED-ALA-003, willing to participate into SPED-ALA-004 study, and meeting any of following criteria. 1) Subject successfully completed treatment period of SPED-ALA-003 2) Subject of which investigator assessed "insufficient effect" during the double blind period of SPED-ALA-003 3) Subject not meeting the transition criteria of double blind period at 24 weeks and general improvement by the investigator was better than "worsening" (2) Subject meeting all eligibility criteria assessed by the investigator (3) 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 (except for study protocol no. SPED-ALA-002 and SPED-ALA-003) (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