UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000059255 |
|---|---|
| Receipt number | R000067778 |
| Scientific Title | Reliability Assessment of Disease-Specific Patient-Reported Outcomes in Gaucher Disease and Their Correlation with Clinical Information |
| Date of disclosure of the study information | 2025/10/01 |
| Last modified on | 2025/10/01 10:25:46 |
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Basic information
Public title
Reliability Assessment of Disease-Specific Patient-Reported Outcomes in Gaucher Disease and Their Correlation with Clinical Information
Acronym
Gaucher Disease-Specific Patient-Reported Outcomes Survey
Scientific Title
Reliability Assessment of Disease-Specific Patient-Reported Outcomes in Gaucher Disease and Their Correlation with Clinical Information
Scientific Title:Acronym
Gaucher Disease-Specific Patient-Reported Outcomes Survey
Region
| Japan |
Condition
Condition
Gaucher disease
Classification by specialty
| Endocrinology and Metabolism | Pediatrics | Nursing |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
We will validate the criterion-related validity and test-retest reliability of patient-reported outcomes (PROs) developed for patients with Gaucher disease, as well as the minimal clinically important distance (MID) in clinical practice.
Basic objectives2
Others
Basic objectives -Others
Using PRO data and clinical data collected by specialists during examinations, we will clarify differences in PROs based on disease type and presence of symptoms.
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Gaucher Disease PRO
Key secondary outcomes
SF-8
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) At the time of consent acquisition, the patient must be 18 years of age or older; for patients under 18 years of age or patients with neurological disease, the consent must be obtained from a caregiver who is 18 years of age or older
2) Gender is not a factor
3) Individuals diagnosed with Gaucher disease
Key exclusion criteria
1) Individuals hospitalized due to acute symptoms
2) Other individuals deemed unsuitable as research subjects by the principal investigator (or co-investigator)
Target sample size
30
Research contact person
Name of lead principal investigator
| 1st name | Yuta |
| Middle name | |
| Last name | Koto |
Organization
Kansai Medical University
Division name
Faculty of Nursing
Zip code
573-1004
Address
2-2-2 Shinmachi, Hirakata city, Osaka, Japan
TEL
072-804-0095
kotou.yuu@kmu.ac.jp
Public contact
Name of contact person
| 1st name | Yuta |
| Middle name | |
| Last name | Koto |
Organization
Kansai Medical University
Division name
Faculty of Nursing
Zip code
573-1004
Address
2-2-2 Shinmachi, Hirakata city, Osaka, Japan
TEL
072-804-0095
Homepage URL
kotou.yuu@kmu.ac.jp
Sponsor or person
Institute
Kansai Medical University
Institute
Department
Personal name
Funding Source
Organization
Japan Society for the Promotion of Science (JSPS), Grants-in-Aid for Scientific Research (KAKENHI)
Organization
Division
Category of Funding Organization
Government offices of other countries
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Kansai Medical University Center for Ethical Review
Address
2-5-1 Shinmachi, Hirakata city, Osaka, Japan
Tel
072-804-0101
rinriirb@hirakata.kmu.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
| 2025 | Year | 10 | Month | 01 | 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
Preinitiation
Date of protocol fixation
| 2025 | Year | 09 | Month | 08 | Day |
Date of IRB
| 2025 | Year | 09 | Month | 11 | Day |
Anticipated trial start date
| 2025 | Year | 10 | Month | 10 | Day |
Last follow-up date
| 2027 | Year | 03 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
This study is a longitudinal questionnaire survey using disease-specific PRO for Gaucher disease. The questionnaire will be distributed and collected repeatedly every four weeks. Furthermore, we will examine the association between data from participants who provided consent for the provision of medical information and their PRO data.
Management information
Registered date
| 2025 | Year | 10 | Month | 01 | Day |
Last modified on
| 2025 | Year | 10 | Month | 01 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000067778
