NCT01828463

Dose Response Study of Nitisinone in Alkaptonuria

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.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (actual)
• Sponsor: University of Liverpool · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Alkaptonuria

Interventions

Type	Name	Description
DRUG	Nitisinone	doses 1, 2, 4 \& 8 mg plus no treatment arm

Timeline

• Start date: 2013-05-01
• Primary completion: 2013-10-01
• Completion: 2013-12-01
• First posted: 2013-04-10
• Last updated: 2014-06-11

Locations

1 site across 1 country: United Kingdom