Development of Patient-Reported and Observer-Reported Outcome Measures for Quality of Life in Patients with Metachromatic Leukodystrophy, Adrenoleukodystrophy, and Krabbe Disease

Development of Patient-Reported and Observer-Reported Outcome Measures for Quality of Life in Patients with Metachromatic Leukodystrophy, Adrenoleukodystrophy, and Krabbe Disease - Development of quality of life scale for leukodystrophy

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000059602

Enrollment

Registered

2025-11-01

Start date

2025-11-01

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

Metachromatic leukoencephalopathy, Adrenoleukodystrophy, Krabbe disease

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1) Individuals aged 18 years or older at the time of consent acquisition; for patients under 18 years of age or those with neurological symptoms or other conditions making self-response difficult, the consent will be obtained from a caregiver aged 18 years or older. 2) Gender is not a factor. 3) Individuals diagnosed with MLD, ALD, or Krabbe disease and their caregivers.

Exclusion criteria

Exclusion criteria: 1) Individuals hospitalized due to acute symptoms 2) Other individuals deemed unsuitable as research subjects by the principal investigator (or co-investigator)

Design outcomes

Primary

Measure	Time frame
Proposed Quality of Life Scale for Leukodystrophy	—

Countries

Japan

Contacts

Public ContactYuta Koto

Kansai Medical University Faculty of Nursing

kotou.yuu@kmu.ac.jp072-804-0095

Outcome results