Multiple system atrophy
Conditions
Interventions
One daily MSA-01 for 48weeks with 300mg dose-escalation tolerable from 300mg up to 1500mg every 2 weeks.
One daily placebo for 48 weeks.
Sponsors
The University of Tokyo Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: [Observation period] 1.Subjects who were diagnosed as probable or possible multiple system atrophy on the basis of the Gilman criteria 2.Subjects who can walk 10 m independently with or without an assistive device including cane, walker, or handrails 3.Subjects who underwent through genetic testing for the detection of mutations in the COQ2 gene 4.Male or female subjects between the ages of 30 and 80 at time of informed consent 5.Subjects who can get an informed consent from themselves 6.Subjects who can be admitted or hospitalized at the medical institution participating in this study The additional features include: <Possible MSA-P or MSA-C> Babinski sign with hyperreflexia Stridor <Possible MSA-P> Rapidly progressive parkinsonism Poor response to levodopa Postural instability within 3 y of motor onset Gait ataxia, cerebellar dysarthria, limb ataxia, or cerebellar oculomotor dysfunction Dysphagia within 5 y of motor onset Atrophy on MRI of putamen, middle cerebellar peduncle, pons, or cerebellum Hypometabolism on FDG-PET in putamen, brainstem, or cerebellum <Possible MSA-C> Parkinsonism (bradykinesia and rigidity) Atrophy on MRI of putamen, middle cerebellar peduncle, or pons Hypometabolism on FDG-PET in putamen Presynaptic nigrostriatal dopaminergic denervation on SPECT or PET [At the beginning of dose-escalation period] 1. The change in total score of unified multiple system atrophy rating scale (UMSARS) part 2 were less than 3 during the observation period 2. Subjects who have not taken the prohibited concomitant medications (see 9.4) 3. No potential clinical risks has been found during the observation period 4. Subjects who are considered adequate to participate in the study by the investigator
Exclusion criteria
Exclusion criteria: 1.Subjects who take medicines, quasi-drugs or supplement contain at least either one of ubiquinone, ubiquinol, idebenone (related products which are prohibited concomitantly), and statins at beginning of the observation period 2.Subjects with severe liver dysfunction (Child-Pugh class B-C) 3.Female subjects who are pregnant, nursing, or possibly pregnant during the trial period (females of childbearing potential are confirmed by either post-menopausal for 3 years prior to the time of informed consent or surgically sterile) 4.Subjects who have received any investigational drug within 3 months prior to informed consent 5.Subjects who are considered inadequate to participate in the study by the investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1)48-weeks progression rate for UMSARS part2 2)Safety (adverse events, vital signs, clinical examination and 12-lead ECG) | — |
Secondary
| Measure | Time frame |
|---|---|
| 1)48-weeks progression rate for UMSARS part2 2)48-weeks progression rate for Barthal index score 3)48-weeks progression rate for SARA score 4)48-weeks progression rate for 10m walking time 5)Progression rate between MSA-C and MSA-P for the primary outcome or the secondary outcome 6)Progression rate between patients with and without COQ2 mutation for the primary outcome or the secondary outcome 7)Coenzyme Q10 levels in plasma | — |
Countries
Japan
Contacts
Public ContactJun Mitsui, M.D., Ph.D.
The University of Tokyo Hospital Department of Neurology
Outcome results
None listed