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

Saitama Medical University Hospital0 个研究点开始时间: 待定最近更新: 2022年8月1日

试验速览

暂无简介。

•Japanese patients who meet all the following criteria (1or2) are included in this clinical trial.
•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
•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.
•Subject successfully completed treatment period of SPED-ALA-003
•Subject of which investigator assessed "insufficient effect" during the double blind period of SPED-ALA-003
•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

Safety

Change in the Newcastle pediatric mitochondrial disease scale (NPMDS) for both central nervous system and neuromuscular symptom(last point of administration from baseline)
The Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Section I~III
NPMDS Section I,II,III, each
NPMDS Section I~IV, totally
Blood FGF21
Body Weight
Body Height
Head circumference
Blood GDF-15
Respiratory chain complex enzymes activity and oxygen consumption in skin fibroblasts
General improvement rate evaluated by Investigator
Brain MRI
Life prognosis
period of ventilator weaning
Number of "stroke-like episodes"

发起方