UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000040117 |
|---|---|
| Receipt number | R000045742 |
| Scientific Title | Physician Awareness and Understanding of Gaucher Disease: A Web based Study |
| Date of disclosure of the study information | 2020/04/13 |
| Last modified on | 2022/06/02 16:08:04 |
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Basic information
Public title
Physician Awareness and
Understanding of Gaucher Disease: A Web based Study
Acronym
Physician Awareness and
Understanding of Gaucher Disease: A Web based Study
Scientific Title
Physician Awareness and
Understanding of Gaucher Disease: A Web based Study
Scientific Title:Acronym
Physician Awareness and
Understanding of Gaucher Disease: A Web based Study
Region
| Japan |
Condition
Condition
Gaucher Disease
Classification by specialty
| Endocrinology and Metabolism |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
This study is designed to answer the following research question:
What is the level of awareness and understanding of GD among physicians?
Basic objectives2
Others
Basic objectives -Others
Web-based questionnaire survey
Trial characteristics_1
Others
Trial characteristics_2
Others
Developmental phase
Not applicable
Assessment
Primary outcomes
- After reading case presentation in the survey, physicians select disease name, his/ her response from list in the survey by visualizing his/ her daily medical practice
- Physicians select his/ her level of awareness, diagnosis experience, available knowledge and interest regarding Gaucher disease.
Key secondary outcomes
Base
Study type
Others,meta-analysis etc
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
Physicians must be:
- Hematologists (including hematologists-oncologists)
- Gastroenterologists
- Currently treating at least 1 in the outpatient or inpatient settings.
Key exclusion criteria
- Gastroenterologists that are not specialized in liver
- Have less than 1 year of practice excluding training period
- Work in a facility other than clinics, university hospitals, general hospitals
and; national and public hospitals
- Do not belong to any society, hematological nor gastroenterological
Target sample size
Research contact person
Name of lead principal investigator
| 1st name | Rieko |
| Middle name | |
| Last name | Sagara |
Organization
Takeda Pharmaceutical Company Limited
Division name
Japan Medical Office
Zip code
1038668
Address
1-1, Nihonbashi-Honcho 2-Chome, Chuo-ku, Tokyo, 103-8668, Japan
TEL
080-7975-2938
rieko.sagara@takeda.com
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
1038668
Address
1-1, Nihonbashi-Honcho 2-Chome, Chuo-ku, Tokyo, 103-8668, Japan
TEL
070-4304-9648
Homepage URL
midori.ono@takeda.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
Clinical research promotion network japan
Address
1-4-9,Itachibori, Nishi-ku, Osaka ,Japan
Tel
06-4393-8403
osaka@sct-net.org
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 | 04 | Month | 13 | Day |
Related information
URL releasing protocol
https://onlinelibrary.wiley.com/doi/10.1111/imj.15790
Publication of results
Published
Result
URL related to results and publications
https://onlinelibrary.wiley.com/doi/10.1111/imj.15790
Number of participants that the trial has enrolled
326
Results
Survey results from 160 haematologists and 166 gastroenterologists indicated that more than 50% of haematologists were aware of GD symptoms, diagnostic criteria and/or treatments, and 38% of them had experienced or suspected GD. The majority of gastroenterologists were unaware of GD or knew the disease only by name, with 20% experiencing or suspecting GD in practice. Almost 70% of haematologists knew of ERT, while 47% of gastroenterologists were not aware of any treatments for GD.
Results date posted
| 2022 | Year | 06 | Month | 02 | Day |
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
160 haematologists
166 gastroenterologists
Both haematologists and gastroenterologists had a mean age around 50 years old, with most having more than 10 years of practical experience.
More than 80% of the respondents had certification from their relevant specialist society.
Most haematologists worked in hospitals with more than 500 beds, and most gastroenterologists worked in clinics with no beds.
Participant flow
After sending the link to the physician panel, the survey website was accessed 673 times. 13 of these responses did not give consent to participate.
In the interest of controlling the reliability of responses, we excluded:
- straight- liners for which all questions in a series had responses in the same location
- responders with logical errors (e.g., inconsistency between age and years of experience)
- fast- responders who completed the survey in less than 1/3 the average time.
Adverse events
Not applicable
Outcome measures
1. Describe the level of awareness of GD in medical institutions
Haematologists had greater practical awareness of GD (symptoms/conditions, diagnostic criteria, treatment methods) than gastroenterologists, who were mostly aware of the disease in name only. Furthermore, 38% of haematologists and 20% of gastroenterologists reported experience suspecting GD in their practice, with even fewer making a diagnosis.
2. Ascertain the level of understanding of GD in medical institutions
Splenomegaly was the most well-known sign from the GED-C, as it was recognized as an indicator of GD by more than 80% of haematologists and 70% of gastroenterologists. By contrast, knowledge of elevated ferritin and hypergammaglobulinemia indicators were low in comparison to other signs included in the GED-C. Furthermore, there were apparent differences between specialties in their explicit awareness of GD signs. Specifically, most haematologists were aware of bone-pain symptoms (68%), whereas less than a third of gastroenterologists (28%) knew of this GD sign.
3. Assess the level of diagnostic understanding of GD in medical institutions
More than 80% of haematologists and 50% of gastroenterologists suspected GD for case 1. On the other hand, when physicians ranked the diseases according to its likeliness, only 28% of haematologists first suspected GD and less than half the gastroenterologists suspected GD in the patient, with most ranking GD among the least likely causes.
An explicit awareness of splenomegaly and bone-pain as signs of GD were significantly predictive of diagnostic accuracy for haematologists according to multiple regression. Splenomegaly was the strongest predictor, and the greatest degree of standard error in prediction accuracy was for responders who did not know the symptoms of GD.
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Completed
Date of protocol fixation
| 2020 | Year | 03 | Month | 30 | Day |
Date of IRB
| 2020 | Year | 04 | Month | 02 | Day |
Anticipated trial start date
| 2020 | Year | 04 | Month | 13 | Day |
Last follow-up date
| 2020 | Year | 04 | Month | 21 | Day |
Date of closure to data entry
| 2020 | Year | 04 | Month | 21 | Day |
Date trial data considered complete
| 2020 | Year | 04 | Month | 24 | Day |
Date analysis concluded
| 2020 | Year | 06 | Month | 02 | Day |
Other
Other related information
Web based survey for physicians
Physicians were recruited through the Nikkei BP Panel (NBP) and participation was subject to inclusion/exclusion criteria for the study. We used a quantitative web-survey comprising a questionnaire of GD awareness and a test of diagnostic accuracy using two fictional model patients. The web survey was conducted between April and May 2020.
Management information
Registered date
| 2020 | Year | 04 | Month | 09 | Day |
Last modified on
| 2022 | Year | 06 | Month | 02 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000045742
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| Research case data specifications | |
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| Registered date | File name |
| Research case data | |
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