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	2024/07/05 09:52:12

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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

Gaucher Disease

Classification by specialty

Endocrinology and Metabolism

Classification by malignancy

Others

Genomic information

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

Email

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

Email

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

Email

osaka@sct-net.org

Secondary IDs

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

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

Main results already published

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

2024 Year 07 Month 05 Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000045742