NCT00432822终止2 期
Double-Blind, Placebo Controlled, Multicentre Study With an Open Label Extension to Evaluate the Efficacy and Safety of Tetrahydrobiopterin (BH4) in Children and Adolescents With Hyperphenylalaninemia Caused by Phenylalanine Hydroxylase Deficiency
Orphanetics Pharma Entwicklungs GmbH0 个研究点目标入组 50 人开始时间: 2007年2月8日最近更新:
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 50
- 主要终点
- dietary phenylalanine tolerance
研究概览
简要总结
The aim of the study is to confirm the efficacy and safety of BH4 in the treatment of hyperphenylalaninemia caused by phenylalanine hydroxylase deficiency in patients responsive to BH4. The primary objective is to assess the effect of BH4 on phenylalanine tolerance compared to placebo under optimal blood phenylalanine control and to demonstrate safety in 12 months long-term treatment. Additionally population PK will be assessed.
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
- Double
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Female and male patients, aged 0-18 years
- •Phenylalanine-4-hydroxylase (PAH) deficiency shown by mutation analysis
- •Blood phenylalanine concentration in the target range under dietary treatment
- •Written consent of a parent or legal representative
- •Assumed availability within the period of study participation
- •Patients/parents willing and able to follow the recommended diet
- •Use of an effective method of contraception in female patients of child bearing potential
排除标准
- •BH4-deficiency due to genetic disorders in biosynthesis or recycling of BH4
- •History or current evidence of poor diet compliance
- •History or current evidence of clinically relevant allergic or idiosyncratic reactions to drugs or food
- •History of allergic reactions to BH4 or its excipients
- •Positive pregnancy test (ß-HCG in serum) and lactating females
- •Participation in other drug trials within the last 30 days before start for the study
结局指标
主要结局
dietary phenylalanine tolerance
safety
次要结局
未报告次要终点
研究者
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