An observational study of health-related quality of life (HRQOL) in patients with inborn errors of metabolism

Not Applicable

• Conditions: Inborn errors of metabolism

• Registration Number: JPRN-UMIN000038674
• Lead Sponsor: Public Health Research Foundation

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-11-25
• Study Completion: 2021-03-31
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Complete: follow-up continuing
• Sex: All
• Target Recruitment: 165

Inclusion Criteria

Not provided

Exclusion Criteria

1. Patients judged by the physician to be inappropriate for registration in this study. 2. Patients unable to comprehend consent form or give consent for themselves except in the case of proxy consent by caregiver.

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Assessment time point: single time point Descriptive statistics of HRQOL values in patients with inborn errors of metabolism

Secondary Outcome Measures

Name	Time	Method
Assessment time point: single time point 1. Evaluation of the correlation between different QOL instruments. 2. Evaluation of the correlation between patient- and caregiver-reported QOL outcomes. 3. Evaluation of the correlation between caregiver burden and patient QOL.