Mitochondrial disease, involved mainly central nervous system
Conditions
Interventions
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 25-75
Sponsors
Saitama Medical University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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
Exclusion criteria
Exclusion criteria: 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety | — |
Secondary
| Measure | Time frame |
|---|---|
| 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) | — |
Countries
Japan
Contacts
Public ContactAkira Otake
Saitama Medical University Hospital
Outcome results
None listed