Dose Response Study of Nitisinone in Alkaptonuria

Phase 2

Completed

• Conditions: Alkaptonuria
• Interventions: Drug: Nitisinone

• Registration Number: NCT01828463
• Lead Sponsor: University of Liverpool

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 Description

Not available

Study Timeline

• Study First Submitted: 2013-04-05
• Study First Submitted QC: 2013-04-05
• Study First Posted: 2013-04-10
• Study Start: 2013-05
• Primary Completion: 2013-10
• Study Completion: 2013-12
• Status Verified: 2014-06
• Last Update Submitted: 2014-06-10
• Last Update Posted: 2014-06-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Nitisinone 1mg	Nitisinone	interventional
Nitisinone 2mg	Nitisinone	interventional
Nitisinone 4mg	Nitisinone	interventional
Nitisinone 8mg	Nitisinone	interventional

Primary Outcome Measures

Name	Time	Method
dose of nitisinone that decreases urinary homogentisic acid to near normal	4 weeks

Trial Locations

Locations (1)

Royal Liverpool Hospital; 🇬🇧 Liverpool, Merseyside, United Kingdom