An International, Multicentre, Randomised, Evaluator-blind, 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.
试验速览
- 阶段
- 3 期
- 入组人数
- 140
- 试验地点
- 1
- 主要终点
- 24 houre Urine Homogentisic acid
研究概览
简要总结
This is a proposal to develop the orphan designated drug, nitisinone, for the treatment of a rare Mendelian disease, Alkaptonuria (AKU). Thanks to our existing successful fundamental and clinical research (cell models, animal models, natural history studies), we are now ready for this final stage of clinical development of nitisinone for AKU: a phase 3 clinical trial to prove efficacy. The results of DevelopAKUre will allow us to make the case to the European Medicines Agency for marketing authorisation of nitisinone for AKU, thereby contributing to the goal of the International Rare Diseases Research Consortium of developing 200 new therapies by 2020.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 25 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A patient must fulfil the following criteria in order to be included in the study:
- •Diagnosis of AKU Any Clinical manifestations of AKU, such as clinical ochronosis or chronic back/joint pain.
- •Age ≥25 years.
- •Willing and able to visit the investigational site for study visits.
- •Signed written informed consent given.
排除标准
- •The presence of any of the following will exclude a patient from inclusion in the study:
- •Currently pregnant or lactating.
- •Female patient of child-bearing potential not using a reliable method of contraception.
- •Known allergy to nitisinone or any of the constituents of the investigational product.
- •Current malignancy.
- •Uncontrolled hypertension (blood pressure greater than 180 mmHg systolic or greater than 95 mmHg diastolic).
- •Unstable cardiovascular disease.
- •Serum potassium < 3.0 mmol/L.
- •eGFR < 60 mL/min .
- •ALT > 1.5 x upper limit of normal.
- •Haemoglobin < 10.0 g/dL.
- •Platelets < 100 x 109/L.
- •Total white blood count < 3.0 x 109/L or neutrophil count < 1.5 x 109/L.
- •History of alcohol or drug abuse.
- •Participation in another clinical study within 3 months of randomization.
- •Treatment with nitisinone within 60 days of randomization.
- •Psychiatric or somatic illness that interferes with compliance or communication with health care personnel.
- •Foreseeable inability to cooperate with given instructions or study procedures.
- •Any other medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion.
研究组 & 干预措施
Nitisinone
Homogentisic acid lowering drug intervention
干预措施: Nitisinone (Drug)
结局指标
主要结局
24 houre Urine Homogentisic acid
时间窗: year 1
次要结局
未报告次要终点
研究者
Professor Lakshminarayan Ranganath
Professor
University of Liverpool