Clinical Trial: Dose Response Study of Nitisinone in Alkaptonuria

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: 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 A

Brief 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.

Detailed Summary:
Sponsor: University of Liverpool

Current Primary Outcome: dose of nitisinone that decreases urinary homogentisic acid to near normal [ Time Frame: 4 weeks ]

Original Primary Outcome: dose of nitisinone that decreases urinary homgenistic acid to near normal [ Time Frame: 4 weeks ]

Current Secondary Outcome:

Original Secondary Outcome:

Information By: University of Liverpool

Dates:
Date Received: April 5, 2013
Date Started: May 2013
Date Completion:
Last Updated: June 10, 2014
Last Verified: June 2014