CompletedPhase 2

Dose Response Study of Nitisinone in Alkaptonuria

United Kingdom40 participantsStarted 2013-05

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Diagnosis of alkaptonuria verified by documented elevated urinary homogentisic acid excretion.
✓. Age ≥18 years.
✓. Willing and able to visit the investigational site for study visits.
✓. Signed written informed consent obtained.

Exclusion criteria

✕. Non-alkaptonuria causes of ochronosis.
✕. Currently pregnant or lactating.
✕. Known allergy to nitisinone or any of the constituents of the investigational product.
✕. Use of a protein-restricted diet
✕. Dietary habits or use of homeopathic therapies that interfere with tyrosine catabolism.
✕. Current keratopathy, contact lens use or uncontrolled glaucoma.
✕. Current malignancy.
✕. Uncontrolled hypertension (blood pressure greater than 180 systolic or greater than 95 diastolic).

What they're measuring

dose of nitisinone that decreases urinary homogentisic acid to near normal

Timeframe: 4 weeks

Trial details

NCT IDNCT01828463

SponsorUniversity of Liverpool

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2013-10

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