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Name:
UMIN ID:

Recruitment status Preinitiation
Unique ID issued by UMIN UMIN000042872
Receipt No. R000048930
Scientific Title Development and evaluation of Gaucher disease (GD) - specific patient reported outcome measurement (PROM) in Japan.
Date of disclosure of the study information 2020/12/29
Last modified on 2020/12/29

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Basic information
Public title Development and evaluation of Gaucher disease (GD) - specific patient reported outcome measurement (PROM) in Japan.
Acronym Development and evaluation of Gaucher disease (GD) - specific patient reported outcome measurement (PROM) in Japan.
Scientific Title Development and evaluation of Gaucher disease (GD) - specific patient reported outcome measurement (PROM) in Japan.
Scientific Title:Acronym Development and evaluation of Gaucher disease (GD) - specific patient reported outcome measurement (PROM) in Japan.
Region
Japan

Condition
Condition Gaucher Disease
Classification by specialty
Endocrinology and Metabolism Pediatrics
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 To develop a Health Related-Quality of Life (HR-QoL) questionnaire specific to all Japanese GD patients.
Basic objectives2 Others
Basic objectives -Others For patients: Restriction of activities, education, and work; Concern about comorbidities, disease burden, and cost; Concerns relating to efficacy and availability of treatment; Context of GD concerns in relation to other medical concerns; how dependent on other people caused by GD; Presence/severity of symptoms; Satisfaction with treatment; Concern about the future
For caregivers:
- social functioning; impact on daily activities; emotional/psychological functioning; physical functioning; and financial impact, burden of caregivers including impact on social life
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes For patients: Restriction of activities, education, and work; Concern about comorbidities, disease burden, and cost; Concerns relating to efficacy and availability of treatment; Context of GD concerns in relation to other medical concerns; how dependent on other people caused by GD; Presence/severity of symptoms; Satisfaction with treatment; Concern about the future
For caregivers:
- social functioning; impact on daily activities; emotional/psychological functioning; physical functioning; and financial impact, burden of caregivers including impact on social life
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 Patients: Subjects aged 16 or above, who are diagnosed and treated for GD
Caregivers: Subjects aged 20 or above, giving care to GD patients
Key exclusion criteria Patients:
- Patients who have cognitive disabilities,
- Patients who lack of fluency in Japanese.
Caregivers:
- Persons with cognitive disabilities
- Persons with lack of fluency in Japanese
Target sample size 100

Research contact person
Name of lead principal investigator
1st name Norio
Middle name
Last name Sakai
Organization Osaka University Graduate School of Medicine
Division name Child Healthcare and Genetic Science Laboratory, Division of Health Science
Zip code 565-0871
Address 1-7, Yamadaoka, Suita-shi, Osaka, Japan
TEL 06-6879-2531
Email norio@sahs.med.osaka-u.ac.jp

Public contact
Name of contact person
1st name Midori
Middle name
Last name Ono
Organization Takeda Pharmaceutical Company Limited
Division name Japan Medical Office
Zip code 6158074
Address 1-1, Nihonbashi-Honcho 2-Chome, Chuo-ku, Tokyo, 103-8668, Japan
TEL 070-4304-9648
Homepage URL
Email mdrwono@gmail.com

Sponsor
Institute Takeda Pharmaceutical Company Limited
Institute
Department

Funding Source
Organization Takeda Pharmaceutical Company Limited
Organization
Division
Category of Funding Organization Profit organization
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization Osaka University Clinical Research Review Committee
Address 2-2, Yamadaoka, Suita, Osaka, Japan
Tel 06-6210-8296
Email rinri@hp-crc.med.osaka-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
2020 Year 12 Month 29 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
2020 Year 11 Month 25 Day
Date of IRB
2020 Year 12 Month 23 Day
Anticipated trial start date
2020 Year 12 Month 28 Day
Last follow-up date
2021 Year 10 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information NA

Management information
Registered date
2020 Year 12 Month 29 Day
Last modified on
2020 Year 12 Month 29 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000048930

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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