UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000056345 |
|---|---|
| Receipt number | R000064369 |
| Scientific Title | A Patient Registry Study of Lysosomal Storage Diseases using the National platform for Rare Disease Data Registry of Japan (Raddar-J) |
| Date of disclosure of the study information | 2024/12/04 |
| Last modified on | 2024/12/06 09:32:43 |
* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments
Basic information
Public title
A Patient Registry Study of Lysosomal Storage Diseases using the National platform for Rare Disease Data Registry of Japan (Raddar-J)
Acronym
A Patient Registry Study of Lysosomal Storage Diseases using the National platform for Rare Disease Data Registry of Japan (Raddar-J)
Scientific Title
A Patient Registry Study of Lysosomal Storage Diseases using the National platform for Rare Disease Data Registry of Japan (Raddar-J)
Scientific Title:Acronym
A Patient Registry Study of Lysosomal Storage Diseases using the National platform for Rare Disease Data Registry of Japan (Raddar-J)
Region
| Japan |
Condition
Condition
Lysosomal Storage Diseases
Classification by specialty
| Endocrinology and Metabolism | Neurology | Pediatrics |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
The primary objective of this study is to establish an observational cohort of patients with lysosomal storage diseases (LSDs) in Japan and follow up the patients for a long period of time to clarify natural history of LSDs, and then to develop a new therapy for these diseases.
Basic objectives2
Others
Basic objectives -Others
An observational cohort of patients with LSDs
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
1) a change in clinical finding over the time
2) lifespan by disease
3) EQ-5D-5L score
4) a change in labo data over the time
5) Common Terminology Criteria for Adverse Events (CTCAE)
Key secondary outcomes
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
| Not applicable |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1) Patients with LSDs confimed by the dianostic criteria certificated by Japanese government
2) Written informed consent
Key exclusion criteria
There are no special exclusion criteria.
Target sample size
10
Research contact person
Name of lead principal investigator
| 1st name | Norio |
| Middle name | |
| Last name | Sakai |
Organization
Iseikai International General Hospital
Division name
Center for Promoting Treatment of Intractable Diseases
Zip code
530-0052
Address
4-14 Minami-ogimachi, Kitaku, Osaka
TEL
81-570-099166
norio@sahs.med.osaka-u.ac.jp
Public contact
Name of contact person
| 1st name | Yasuyuki |
| Middle name | |
| Last name | Fukuhara |
Organization
National Center for Child Health and Development
Division name
Division of medical genetics
Zip code
157-8535
Address
2-10-1, Okura, Setagaya-ku, Tokyo
TEL
81-3-3416-0181
Homepage URL
fukuhara-ys@ncchd.go.jp
Sponsor or person
Institute
Iseikai International General Hospital
Institute
Department
Personal name
Funding Source
Organization
Ministry of Health, Labour and Welfare
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
Kyoto University Graduate School and Faculty of Medicine, Ethics Committee
Address
Yoshida-Konoe-cho, Sakyo-ku, Kyoto 606-8501, JAPAN
Tel
+81-75-753-4680
ethcom@kuhp.kyoto-u.ac.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
Other administrative information
Date of disclosure of the study information
| 2024 | Year | 12 | Month | 04 | 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
Open public recruiting
Date of protocol fixation
| 2023 | Year | 10 | Month | 11 | Day |
Date of IRB
| 2023 | Year | 10 | Month | 11 | Day |
Anticipated trial start date
| 2023 | Year | 11 | Month | 01 | Day |
Last follow-up date
| 2026 | Year | 10 | Month | 30 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
an obsevational cohort study
Management information
Registered date
| 2024 | Year | 12 | Month | 03 | Day |
Last modified on
| 2024 | Year | 12 | Month | 06 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000064369
