Questionnaire Follow=up Study Sonia 2

Completed

• Conditions: Alkaptonuria; Alkaptonuria Severity Score Index; Homogentisate Dioxygenase; Homogentisic Acid

• Registration Number: NCT04510142
• Lead Sponsor: Liverpool University Hospitals NHS Foundation Trust

Brief Summary

Survey of health of patients completing SONIA 2 study: A cross-sectional analysis by questionnaire

Detailed Description

The Liverpool site will post the once only, short questionnaire (in the correct language using translated documents as appropriate) to the address of those patients who completed SONIA 2. Hospital approved translation services will be used. A return-addressed stamped envelope for ease of return will also be provided. Those who received nitisinone will be sent the nitisinone-administration questionnaire and those who did not receive nitisinone will be sent a no-nitisinone questionnaire

Study Timeline

• Study Start: 2019-09-19
• Study First Submitted: 2020-08-10
• Study First Submitted QC: 2020-08-10
• Study First Posted: 2020-08-12
• Primary Completion: 2023-08-31
• Study Completion: 2023-08-31
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-21
• Last Update Posted: 2025-03-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

• Documented diagnosis of AKU. Age ≥16 years.

Exclusion Criteria

• Not completed SONIA 2 study for the full 4-year duration.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
to collect information on their health status since completing participation in SONIA 2	12 months	All of the SONIA 2 completed questionnaire data collection is completed by 2 Sept 2020

Trial Locations

Locations (1)

Liverpool Royal Hospitals NHS Foundation Trust; 🇬🇧 Liverpool, United Kingdom