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. - SONIA 1

niversity of Liverpool (UniLiv)0 个研究点目标入组 40 人开始时间: 2013年1月9日最近更新: 2019年2月28日

研究概览

暂无简介。

•1. Diagnosis of AKU verified by previously documented elevated urinary homogentisic acid excretion.
•2. Age =18 years.
•3. Willing and able to visit the investigational site for study visits.
•4. Signed written informed consent given.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 37
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 3

•1. Currently pregnant or lactating.
•2. Female patient of child-bearing potential not using a reliable method of contraception.
•3. Known allergy to nitisinone or any of the constituents of the investigational product.
•4. Current keratopathy or uncontrolled glaucoma.
•5. Current malignancy.
•6. Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).
•7. Unstable cardiovascular disease.
•8. Serum potassium < 3.0 mmol/L.
•9. eGFR < 60 mL/min.
•10. ALT > 3 x upper limit of normal.
•11. Hemoglobin < 10.0 g/dL.
•12. Platelets < 100 x 109/L.
•13. White blood count < 3.0 x 109/L.
•14. History of alcohol or drug abuse.
•15. Participation in another clinical study within 3 months of randomization.
•16. Treatment with nitisinone within 60 days of randomization.
•17. Psychiatric illness or neurological disease that interferes with compliance or communication with health care personnel.
•18. Any other medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.
•19. Foreseeable inability to cooperate with given instructions or study procedures.

发起方

niversity of Liverpool (UniLiv)