Development and Validation of a Standardized Assessment Instrument for Health-related Quality of Life (HrQoL) in Patients With Classical Homocystinuria

University Children's Hospital, Zurich1 site in 1 country80 target enrollmentJanuary 15, 2025

ConditionsHomocystinuria

Overview

Phase: Not Applicable
Intervention: Not specified
Conditions: Homocystinuria
Sponsor: University Children's Hospital, Zurich
Enrollment: 80
Locations: 1
Primary Endpoint: HrQol
Status: Recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

Brief Summary

Patients, parents of young / handicapped patients, and experts will be interviewed to collect contents relevant for HrQoL in CBS deficiency. Based on these data, a questionnaire will be developed and tested for comprehensibility in patients and parents of young / handicapped patients. A final questionnaire version will be tested for psychometric criteria including validity and reliability.

Registry

clinicaltrials.gov

Start Date

January 15, 2025

End Date

September 2025

Last Updated

last year

Study Type

Observational

Sex

All