Overview

Dose Response Study of Nitisinone in Alkaptonuria

Status:
Completed
Trial end date:
2013-12-01
Target enrollment:
Participant gender:
Summary
SONIA 1 is an international, multicentre, randomised, open-label, no-treatment controlled, parallel group, dose-response study to investigate the effect of once daily nitisinone on 24-hour urinary homogentisic acid excretion in patients with alkaptonuria after 4-weeks treatment. They study will identify the optimal dose to decrease urine homogentisic acid to near normal levels.
Phase:
Phase 2
Details
Lead Sponsor:
University of Liverpool
Collaborators:
Liverpool University Hospitals NHS Foundation Trust
Royal Liverpool and Broadgreen University Hospitals NHS Trust
Treatments:
Nitisinone