UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000042872 |
|---|---|
| Receipt number | 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 | 2022/09/06 14:31:25 |
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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
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
mdrwono@gmail.com
Sponsor or person
Institute
Takeda Pharmaceutical Company Limited
Institute
Department
Personal name
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
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
https://doi.org/10.1186/s13023-022-02429-z
Publication of results
Published
Result
URL related to results and publications
https://doi.org/10.1186/s13023-022-02429-z
Number of participants that the trial has enrolled
8
Results
Three main themes emerged from qualitative analysis of patient interviews: treatment status, patient burden, and social support systems. Commonly used words and the relationships between words identified through the hierarchical cluster and co-occurrence network analyses supported these themes and topics. These themes and topics were specific to patients with types 2 and 3 GD and will be used to inform the development of a HRQOL questionnaire specifically for patients with all GD types
Results date posted
| 2022 | Year | 09 | Month | 06 | Day |
Results Delayed
Results Delay Reason
Date of the first journal publication of results
| 2022 | Year | 07 | Month | 19 | Day |
Baseline Characteristics
Eight patients (six male, two female) recruited from a patient association participated in the interview: four with type 2 GD (four caregivers) and four with type 3 GD (two patients and two caregivers) . All four patients with type 2 GD were 2-11 years old, whereas two patients with type 3 GD were adults and two were < 10 years old.
Participant flow
Participants were recruited by referral from a patient association in Japan (Association of Gaucher Disease Patients in Japan).
Adverse events
NA
Outcome measures
Three main themes emerged from qualitative analysis of patient interviews.:
1.Treatment status
Qualitative analysis of the verbatim transcripts revealed seven main topics related to treatment status that were commonly mentioned by patients and/or caregivers. These topics focused on the impact of physical symptoms of GD on daily life rather than the treatments themselves and included hearing impairment, visual impairment, difficulty swallowing, difficulty speaking, involuntary movement of extremities, epileptic seizures, and body aches.
2.Patient burden
Five main topics emerged as common features of patient burden: anxiety about symptoms, difficulty with exercise and work, anxiety about continuing treatment, anxiety about going out, and tiredness from hospital visits or treatment. Several of the "patient burden" topics overlapped with "treatment status" topics, for example, anxiety about having an epileptic seizure. Indeed, four of the five topics were related to anxiety about various aspects of daily life, including worries about having seizures, particularly during school, work, or exercise, and the need for ongoing treatment.
3.Social support systems
Topics within the "social support systems" theme included dissatisfaction about government services, lack of social support, and information exchange in the patient association.
Several patients indicated that the government services were inadequate and explaining GD to these services was burdensome. Although many patients said that information about social support systems was not available to them, some noted that they were able to exchange information with other patients through patient associations.
Plan to share IPD
NA
IPD sharing Plan description
NA
Progress
Recruitment status
Completed
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
| 2022 | Year | 09 | Month | 06 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000048930
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