Dose Response Study of Nitisinone in Alkaptonuria

Authors
Category Primary study
Registry of Trialsclinicaltrials.gov
Year 2013
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.
Epistemonikos ID: cd92f6675d2f267065c99d297b3d8ebf4be44794
First added on: May 11, 2024