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Name:
UMIN ID:

Recruitment status No longer recruiting
Unique ID issued by UMIN UMIN000031771
Receipt No. R000036134
Scientific Title A Randomized, Double-Blind, Placebo-Controlled, Multicenter study to Evaluate Efficacy and Safety of MSA-01 in Patients with Multiple system atrophy.
Date of disclosure of the study information 2018/03/16
Last modified on 2019/06/05

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Basic information
Public title A Randomized, Double-Blind, Placebo-Controlled, Multicenter study to Evaluate Efficacy and Safety of MSA-01 in Patients with Multiple system atrophy.
Acronym A Randomized, Double-Blind, Placebo-Controlled, Multicenter study to Evaluate Efficacy and Safety of MSA-01 in Patients with Multiple system atrophy.
Scientific Title A Randomized, Double-Blind, Placebo-Controlled, Multicenter study to Evaluate Efficacy and Safety of MSA-01 in Patients with Multiple system atrophy.
Scientific Title:Acronym A Randomized, Double-Blind, Placebo-Controlled, Multicenter study to Evaluate Efficacy and Safety of MSA-01 in Patients with Multiple system atrophy.
Region
Japan

Condition
Condition Multiple system atrophy
Classification by specialty
Neurology
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 To assess the efficacy and the safety of high-dose MSA-01 supplementation in patients with multiple system atrophy under the randomized, double-blind, placebo-controlled study.
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1 Exploratory
Trial characteristics_2
Developmental phase Phase II

Assessment
Primary outcomes 1)48-weeks progression rate for UMSARS part2
2)Safety (adverse events, vital signs, clinical examination and 12-lead ECG)
Key secondary outcomes 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

Base
Study type Interventional

Study design
Basic design Parallel
Randomization Randomized
Randomization unit Individual
Blinding Double blind -all involved are blinded
Control Placebo
Stratification YES
Dynamic allocation NO
Institution consideration Institution is not considered as adjustment factor.
Blocking YES
Concealment Central registration

Intervention
No. of arms 2
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 One daily MSA-01 for 48weeks with 300mg dose-escalation tolerable from 300mg up to 1500mg every 2 weeks.
Interventions/Control_2 One daily placebo for 48 weeks.
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit
30 years-old <=
Age-upper limit
80 years-old >
Gender Male and Female
Key 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
Key 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
Target sample size 120

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Shoji Tsuji, M.D., Ph.D.
Organization The University of Tokyo Hospital
Division name Department of Neurology
Zip code
Address 7-3-1 Hongo, Bunkyo-ku, Tokyo, 113-8655, Japan
TEL 03-3815-5411
Email tsuji@m.u-tokyo.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Jun Mitsui, M.D., Ph.D.
Organization The University of Tokyo Hospital
Division name Department of Neurology
Zip code
Address 7-3-1 Hongo, Bunkyo-ku, Tokyo, 113-8655, Japan
TEL 03-3815-5411
Homepage URL
Email mituij-tky@umin.ac.jp

Sponsor
Institute The University of Tokyo Hospital
Institute
Department

Funding Source
Organization Japan Agency of Medical Research and Development
Organization
Division
Category of Funding Organization Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
Secondary IDs NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions 国立大学法人北海道大学病院(北海道)
新潟大学医歯学総合病院(新潟県)
独立行政法人国立病院機構東埼玉病院(埼玉県)
国立研究開発法人国立精神・神経医療研究センター(東京都)
千葉大学医学部附属病院(千葉県)
国立大学法人東京医科歯科大学医学部附属病院(東京都)
名古屋大学医学部附属病院(愛知県)
京都大学医学部附属病院(京都府)
鳥取大学医学部附属病院(鳥取県)
岡山大学病院(岡山県)
九州大学病院(福岡県)
鹿児島大学病院(鹿児島県)




Other administrative information
Date of disclosure of the study information
2018 Year 03 Month 16 Day

Related information
URL releasing protocol
Publication of results Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status No longer recruiting
Date of protocol fixation
2017 Year 12 Month 19 Day
Date of IRB
2017 Year 12 Month 19 Day
Anticipated trial start date
2018 Year 05 Month 02 Day
Last follow-up date
2020 Year 06 Month 30 Day
Date of closure to data entry
2020 Year 10 Month 31 Day
Date trial data considered complete
2020 Year 10 Month 31 Day
Date analysis concluded
2020 Year 12 Month 31 Day

Other
Other related information

Management information
Registered date
2018 Year 03 Month 16 Day
Last modified on
2019 Year 06 Month 05 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000036134

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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