An international, multicenter, randomized, evaluator-blinded, no-treatment controlled, parallel-group study to assess the efficacy and safety of once daily nitisinone in patients with alkaptonuria after 12 months of treatment, followed by an additional 36-month treatment period.

niversity of Liverpool (UniLiv)0 个研究点目标入组 140 人开始时间: 2013年10月14日最近更新: 2019年6月30日

研究概览

暂无简介。

•1. Diagnosis of AKU.
•2. Any clinical manifestations of AKU, such as clinical ochronosis or chronic back / joint pain.
•3. Age =25 years.
•4. Willing and able to visit the investigational site for study visits.
•5. 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 140
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Treatment with nitisinone within 3 months of randomization.
•2. Participation in another clinical study within 3 months of randomization.
•3. Known allergy to nitisinone or any of the constituents of the investigational product.
•4. Female patient of child-bearing potential not using a reliable method of contraception.
•5. Currently pregnant or lactating.
•6. Current malignancy.
•7. Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).
•8. Unstable cardiovascular disease.
•9. Clinically relevant lab abnormalities.
•10. History of alcohol or drug abuse.
•11. Psychiatric or somatic illness that interferes with compliance or communication with health care personnel.
•12. Foreseeable inability to cooperate with given instructions or study procedures.
•13. Any other medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.

发起方

niversity of Liverpool (UniLiv)