UMIN-ICDS Clinical Trial
| Unique ID issued by UMIN | UMIN000059546 |
|---|---|
| Receipt number | R000068113 |
| Scientific Title | Natural History Study of Central Nervous System Function in Myotonic Dystrophy |
| Date of disclosure of the study information | 2025/10/27 |
| Last modified on | 2025/10/27 12:13:56 |
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Basic information
Public title
Longitudinal Study of Central Nervous System Function in Myotonic Dystrophy
Acronym
Longitudinal Study of Central Nervous System Function in Myotonic Dystrophy
Scientific Title
Natural History Study of Central Nervous System Function in Myotonic Dystrophy
Scientific Title:Acronym
Natural History Study of Central Nervous System Function in Myotonic Dystrophy
Region
| Japan |
Condition
Condition
Myotonic Dystrophy
Classification by specialty
| Neurology |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To evaluate the longitudinal changes in cognitive function in patients with myotonic dystrophy after 10 years from the baseline.
Basic objectives2
Others
Basic objectives -Others
Descriptive
Trial characteristics_1
Exploratory
Trial characteristics_2
Pragmatic
Developmental phase
Not applicable
Assessment
Primary outcomes
The course of change in cognitive function test scores at baseline, 5 years later, and 10 years later
Key secondary outcomes
The course of Changes in PRO scores measured by questionnaires, such as the QoL questionnaire, at baseline, 5 years, and 10 years later.
Time of death after the initial assessment.
Apathy scale
Fatigue Severity Scale
Epworth Sleepiness Scale
Individualized Neuromuscular Quality of Life Questionnaire
Myotonic Dystrophy Health Index
Base
Study type
Observational
Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
Interventions/Control_2
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
| 18 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
This study will include patients aged 18 years or older with confirmed diagnosis of DM1.
Patients must meet all of the following criteria:
1) Patients diagnosed with DM1 based on genetic testing or clinical symptoms and family history
2) Patients who participated in a previous longitudinal study and have available information regarding cognitive assessment
Key exclusion criteria
Patients with congenital conditions, severe symptoms, severe visual impairment, severe hearing impairment, or other factors making research participation difficult will be excluded.
Patients meeting any of the following criteria will not be enrolled in this study:
1) Congenital DM1
2) Severe symptoms, visual impairment, or hearing impairment making research participation difficult
3) If the principal investigator or co-investigator determines the patient is not suitable for participation in this study
Target sample size
67
Research contact person
Name of lead principal investigator
| 1st name | Tsuyoshi |
| Middle name | |
| Last name | Matsumura |
Organization
NHO Osaka Toneyama Medical Center
Division name
Neurology
Zip code
560-8552
Address
Toneyama 5-1-1, Toyonaka, Osaka
TEL
06-6853-2001
410-rinshokenkyu@mail.hosp.go.jp
Public contact
Name of contact person
| 1st name | Haruo |
| Middle name | |
| Last name | Fujino |
Organization
The University of Osaka
Division name
Graduate School of Human Sciences
Zip code
5650871
Address
Yamadaoka 1-2, Suita
TEL
06-6879-8139
Homepage URL
fujino.haruo.hus@osaka-u.ac.jp
Sponsor or person
Institute
NHO Osaka Toneyama Medical Center
Institute
Department
Personal name
Funding Source
Organization
Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization
Japanese Governmental office
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Name of secondary funder(s)
Ministry of Health, Labour and Welfare
IRB Contact (For public release)
Organization
NHO Osaka Toneyama Medical Centre Ethical Review Board
Address
Toneyama 5-1-1, Toyonaka
Tel
06-6853-2001
410-rinshokenkyu@mail.hosp.go.jp
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
大阪大学（大阪府）、あきた病院（秋田県）、沖縄病院（沖縄県）、横浜労災病院（神奈川県）
The University of Osaka, NHO Akita Hospital, NHO Okinawa Hospital, Yokohama Rosai Hospital
Other administrative information
Date of disclosure of the study information
| 2025 | Year | 10 | Month | 27 | 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
Enrolling by invitation
Date of protocol fixation
| 2025 | Year | 07 | Month | 27 | Day |
Date of IRB
| 2025 | Year | 09 | Month | 08 | Day |
Anticipated trial start date
| 2025 | Year | 10 | Month | 09 | Day |
Last follow-up date
| 2027 | Year | 09 | Month | 30 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Observational study, exploratory
Management information
Registered date
| 2025 | Year | 10 | Month | 27 | Day |
Last modified on
| 2025 | Year | 10 | Month | 27 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000068113
