UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000059079 |
|---|---|
| Receipt number | R000067438 |
| Scientific Title | The Burden on Daily Life and Quality of Life for Patients with Mucopolysaccharidosis: An Interview Research |
| Date of disclosure of the study information | 2025/09/16 |
| Last modified on | 2025/09/12 20:50:42 |
* 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
The Burden on Daily Life and Quality of Life for Patients with Mucopolysaccharidosis: An Interview Research
Acronym
Mucopolysaccharidosis Interview Research
Scientific Title
The Burden on Daily Life and Quality of Life for Patients with Mucopolysaccharidosis: An Interview Research
Scientific Title:Acronym
Mucopolysaccharidosis Interview Research
Region
| Japan |
Condition
Condition
Mucopolysaccharidosis
Classification by specialty
| Endocrinology and Metabolism | Pediatrics | Nursing |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To clarify the daily burdens and actual state of quality of life experienced by patients with mucopolysaccharidosis and their families.
Basic objectives2
Others
Basic objectives -Others
To consider candidate questions for the QOL scale.
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Verbatim Transcript of the Interview
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
| 16 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
Patients
1) Patients aged 18 years or older diagnosed with a type of mucopolysaccharidosis without intellectual disability (diagnosed by developmental testing or genetic testing)
2) Patients aged 16 years or older but under 18 years of age diagnosed with a type of mucopolysaccharidosis without intellectual disability (diagnosed via developmental assessment or genetic testing), where both the patient and guardian have provided written consent for study participation (Since the patient is a minor, consent must be obtained from both the patient and guardian. If the guardian wishes to participate in the study, they may attend the interview).
3) No gender restrictions
4) Individuals who have received sufficient explanation regarding the purpose and content of this study and have provided written consent to participate
5) Individuals capable of communicating in Japanese
6) Individuals able to participate in online or in-person interviews
Guardian
1) Guardian of a patient diagnosed with mucopolysaccharidosis, regardless of the patient's age or presence of intellectual disability
2) No gender restrictions
3) Individuals who have received a thorough explanation of the purpose and content of this study and have provided written consent for research participation
4) Individuals capable of communicating in Japanese
5) Individuals able to participate in online or in-person interviews
Key exclusion criteria
Patients
1) Individuals deemed unable to participate in the interview survey due to severe physical complications
2) Other individuals deemed unsuitable as research subjects by the principal investigator (or co-investigator)
Guardian
1) Individuals whose patient is undergoing acute-phase treatment such as hospitalization due to severe physical complications and is deemed unable to participate in the interview survey
2) Other individuals deemed unsuitable as research subjects by the principal investigator (or co-investigator)
Target sample size
20
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, 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, 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 | 09 | 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
Preinitiation
Date of protocol fixation
| 2025 | Year | 09 | Month | 04 | Day |
Date of IRB
| 2025 | Year | 09 | Month | 11 | Day |
Anticipated trial start date
| 2025 | Year | 09 | Month | 16 | Day |
Last follow-up date
| 2028 | 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 employs a qualitative descriptive research design and will conduct interview surveys using a semi-structured interview method. Interviews will be conducted based on an interview guide. Interview content will be recorded, and the recorded data will be transcribed verbatim. The data will then be analyzed using qualitative content analysis.
Management information
Registered date
| 2025 | Year | 09 | Month | 12 | Day |
Last modified on
| 2025 | Year | 09 | Month | 12 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000067438
