臨床研究等提出・公開システム

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

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

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

+81-49-276-1218

akira_oh@saitama-med.ac.jp

COMPLETED

Feb. 01, 2019

Interventional

Unblinded, uncontrolled, long-term, multicenter trial

open(masking not used)

No

3

Japanese 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

Patients 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

Both

Mitochondrial disease, involved mainly central nervous system

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 HCI 25-75and SFC 39.22-117.66 mg Dosing frequency / Frequency of use:BID Dose of investigational drug is determined based on the body weight at each study visit. 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 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. 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:-

Safety

1.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)

Nov. 27, 2018