Development and evaluation of Gaucher disease (GD) - specific patient reported outcome measurement (PROM) in Japan.

Development and evaluation of Gaucher disease (GD) - specific patient reported outcome measurement (PROM) in Japan. - Development and evaluation of Gaucher disease (GD) - specific patient reported outcome measurement (PROM) in Japan.

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000042872

Enrollment

100

Registered

2020-12-29

Start date

2020-12-28

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Gaucher Disease

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

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

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
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	—

Measure

Time frame

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

—

Countries

Japan

Contacts

Public ContactMidori Ono

Takeda Pharmaceutical Company Limited Japan Medical Office

mdrwono@gmail.com070-4304-9648

Outcome results