UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000045622 |
|---|---|
| Receipt number | R000052086 |
| Scientific Title | A Patient-Reported Outcomes Study in Patients with Primary Immunodeficiency in Japan: A Non-interventional Prospective Observational Study |
| Date of disclosure of the study information | 2021/10/29 |
| Last modified on | 2025/05/16 10:47:01 |
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Basic information
Public title
A Patient-Reported Outcomes Study in Patients with Primary Immunodeficiency in Japan:
A Non-interventional Prospective Observational Study
Acronym
PID PRO study in Japan
Scientific Title
A Patient-Reported Outcomes Study in Patients with Primary Immunodeficiency in Japan:
A Non-interventional Prospective Observational Study
Scientific Title:Acronym
PID PRO study in Japan
Region
| Japan |
Condition
Condition
Primary immunodeficiency
Classification by specialty
| Medicine in general | Gastroenterology | Hepato-biliary-pancreatic medicine |
| Pneumology | Hematology and clinical oncology | Clinical immunology |
| Pediatrics | Ophthalmology | Dermatology |
| Oto-rhino-laryngology | Orthopedics | Adult |
| Child |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To assess the HR-QOL of patients with PID
Basic objectives2
Others
Basic objectives -Others
A Non-interventional Prospective Observational Study
Trial characteristics_1
Exploratory
Trial characteristics_2
Pragmatic
Developmental phase
Not applicable
Assessment
Primary outcomes
HR-QOL of patients with PID as assessed by the EuroQol 5 dimensions 5-level (EQ-5D-5L) and Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36)
Key secondary outcomes
-HR-QOL of patients with PID as assessed by the EQ-5D-5L and SF-36, compared with population norms or values recorded in healthy volunteers
-Daily health status of patients with PID compared with that of healthy volunteers
-Assessment of Work Productivity and Activity Impairment Questionnaire (WPAI) and limitations of daily activities in patients with PID
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
| 12 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male and Female
Key inclusion criteria
1) Criteria common to patients with PID and healthy volunteers:
(1) Aged at least 12 years when giving informed consent or assent
(2) Has signed (or if applicable, a legal representative has signed) an informed consent form before undergoing any study procedures
(3) Lives in Japan (at least for the study duration)
(4) Possesses a smartphone (or a minor who does not possess a smartphone but whose legal representative does)
2) Criteria specific to patients with PID:
Those who meet the first two or the first and third of the following requirements:
(1) Patient who has been diagnosed with PID
(2) Patient who is registered at or referred from the non-profit organization (NPO) PID Tsubasa No Kai (patients association)
(3) Patient who is considered eligible for the study and referred for inclusion in the study by a physician who is affiliated with the Japanese Society for Immunodeficiency and Autoinflammatory Disease and approved by the Study Director
3) Criteria specific to healthy volunteers:
None
Key exclusion criteria
1) Criteria common to patients with PID and healthy volunteers:
(1) Works in the pharmaceutical, medical, or research industry (or has a cohabiting family member who does)
(2) Is expected to be unable to answer questions about the specified study variables
2) Criteria specific to patients with PID:
(1) Patients who have been diagnosed with an inherited autoinflammatory disease, such as familial Mediterranean fever
3) Criteria specific to healthy volunteers:
(1) Has type 2 diabetes, hypertension, dyslipidemia, liver disease (hepatitis), renal disease, cardiac disease, thyroid disease, adrenal disease, any other metabolic disorder, or any mental disease such as depression at the time of giving informed consent (and assent, if applicable)
(2) Has any physical symptom that will interfere with his/her daily life at the time of giving informed consent (and assent, if applicable)
(3) Is regularly taking medicine or attending a clinic for any disease (seasonal allergic diseases such as pollinosis and any medication/clinic visits to treat the disease are excluded) at the time of giving informed consent (and assent, if applicable)
(4) Has a history of any severe medical condition
(5) Has a drug or food allergy
(6) Has a family history of PID
Target sample size
200
Research contact person
Name of lead principal investigator
| 1st name | Kaori |
| Middle name | |
| Last name | Nakagawa |
Organization
Takeda Pharmaceutical Company Limited.
Division name
Japan Medical Office
Zip code
103-8668
Address
1-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo
TEL
070-3062-6158
kaori.nakagawa@takeda.com
Public contact
Name of contact person
| 1st name | Madoka |
| Middle name | |
| Last name | Go |
Organization
Takeda Pharmaceutical Company Limited.
Division name
Japan Medical Office
Zip code
103-8668
Address
1-1, Nihonbashi-Honcho 2-chome, Chuo-ku, Tokyo
TEL
090-1872-0300
Homepage URL
madoka.go@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
Japan
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Japan Conference of Clinical research
Address
1-13-23 Minamiikebukuro, Toshima-ku, Tokyo
Tel
03-6868-7022
jccr-info@jccr.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
該当せず（NA）
Other administrative information
Date of disclosure of the study information
| 2021 | Year | 10 | Month | 29 | Day |
Related information
URL releasing protocol
https://link.springer.com/article/10.1007/s10875-025-01869-z
Publication of results
Published
Result
URL related to results and publications
https://link.springer.com/article/10.1007/s10875-025-01869-z
Number of participants that the trial has enrolled
127
Results
Patients with PID had a lower HR-QOL all year round and were more socially, physically, and mentally stressed in all seasons compared with healthy people.
Results date posted
| 2025 | Year | 05 | Month | 16 | Day |
Results Delayed
Results Delay Reason
Date of the first journal publication of results
| 2025 | Year | 05 | Month | 07 | Day |
Baseline Characteristics
- Patients with PID
- Healthy volunteers
Participant flow
In this study, leaflets were distributed through a patient association and multiple medical institutions. Participants accessed the website provided on the leaflet and completed the consent process and questionnaire online.
Adverse events
N/A
Outcome measures
Main items
- EuroQol-5 Dimensions-5 Levels (EQ-5D-5L)
- Medical Outcomes Study 36-Item Short Form Health Survey (SF-36)
- Work Productivity and Activity Impairment (WPAI)
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Main results already published
Date of protocol fixation
| 2021 | Year | 09 | Month | 17 | Day |
Date of IRB
| 2021 | Year | 10 | Month | 22 | Day |
Anticipated trial start date
| 2021 | Year | 11 | Month | 01 | Day |
Last follow-up date
| 2023 | Year | 05 | Month | 19 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
A Non-interventional Prospective Observational Study(PRO study) in Patients with Primary Immunodeficiency in Japan:
Management information
Registered date
| 2021 | Year | 10 | Month | 01 | Day |
Last modified on
| 2025 | Year | 05 | Month | 16 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000052086
