EUCTR2006-000648-15-AT进行中（未招募）不适用
A 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 75 人开始时间: 2006年4月5日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 75
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Inclusion criteria for the screening phase
- •1.Female and male patients, aged 4-18 years
- •2.Phenylalanine-4-hydroxylase (PAH) deficiency shown by mutation analysis
- •3.Blood phenylalanine concentration in the 42-240 µmol/L for patients <10yrs otherwise 42-360 µmol/L range under dietary treatment
- •4.Achievement of phenylalanine equilibrium for at least 1 week before the screening phase
- •5.Written consent of patient (if only enough understand) and a parent or legal representative of the paediatric or adolescent patient after being given oral and written information on the study
- •6.Informed consent of the patient if she/he is able to understand the advantages/disadvantages of the participation in the study (this can be expected at a minimum age of 8 years). Written consent of an adolescent patient after oral and written information or oral consent procedure with an impartial witness is acceptable.
- •7.Assumed availability within the period of study participation
- •8.Patients/parents willing and able to follow the recommended diet
- •9.Use of an effective method of contraception in female patients of child bearing potential
- •Inclusion criteria for the treatment phase
- •1.BH4-responsiveness determined by a combined phenylalanine-BH4 test (see Section 7.5.2 Combined phenylalanine-BH4-loading test)
- •2.Achievement of phenylalanine intake equilibrium for at least 1 week
- •with subsequent determination of baseline phenylalanine tolerance (Phe-Tol0)
- •Are the trial subjects under 18? yes
- •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
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
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
- •?History or current evidence of any clinically relevant cardiovascular, pulmonary, renal, gastrointestinal, haematological, endocrinological, metabolic, neurological or other diseases as judged by the investigator
- •?History of malignancy within the past 5 years
- •?Relevant chronic or relevant current acute infections
- •?History of orthostatic deregulation, fainting or blackout
- •?Abuse of alcohol or drugs
- •?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
- •?Patient already receiving BH4 treatment or who has received it within the last 1 month
研究者
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