Primary immunodeficiency
Conditions
Interventions
None listed
Sponsors
Takeda Pharmaceutical Company Limited.
Eligibility
Sex/Gender
All
Inclusion criteria
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
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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) | — |
Secondary
| Measure | Time frame |
|---|---|
| -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 | — |
Countries
Japan
Contacts
Public ContactMadoka Go
Takeda Pharmaceutical Company Limited. Japan Medical Office
Outcome results
None listed