NCT01965912已完成4 期
A Phase 4 Open-Label, Single-Cohort Study of the Long-Term Neurocognitive Outcomes in 4 to 5 Year-Old Children With Phenylketonuria Treated With Sapropterin Dihydrochloride (Kuvan®) for 7 Years
适应症
干预措施
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 34
- 试验地点
- 6
- 主要终点
- Mean Full Scale Intelligence Quotient (FSIQ) Score of the Wechsler Intelligence Scale for Children (WISC)-IV
研究概览
简要总结
The study is a single-cohort, interventional, open-label trial to evaluate long-term neurocognitive (NC) outcomes in children aged 4 to 5 years with phenylketonuria (PKU) treated with Kuvan® and Phenylalanine-restricted diet over a period of 7 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female outpatients, 4 to 5 years of age (greater than or equal to 4 and less than 6) at the time of informed consent form signature by parent(s) or guardian(s)
- •Confirmed clinical and biochemical diagnosis of PKU, including at least two separate blood phenylalanine levels greater than or equal to 400 micromole per liter (mcmol/L)
- •Defined pre-Kuvan®/-tetrahydrobiopterin (BH4) dietary phenylalanine tolerance consistent with the diagnosis of PKU
- •Responsive to Kuvan®/BH4:
- •For subjects currently treated with Kuvan®/BH4 at Screening: subject is a responder as per Investigator judgment based on documented effect of Kuvan®/BH4 on phenylalanine levels and/or phenylalanine tolerance
- •For subjects not treated with Kuvan®/BH4 at Screening: a response test has been performed during Screening or is available from the subject's medical records and satisfies the 3 following criteria: a decrease in blood phenylalanine levels of at least 30 percent was observed after at least 24 hours with a dose of at least 10 mg/kg/day
- •Intelligence Quotient (IQ) greater than or equal to 70, as assessed with the Wechsler Preschool and Primary Scale of Intelligence (WPPSI)-III, 2nd part
- •Good adherence with dietary treatment (including prescribed dietary phenylalanine restriction and prescribed amounts of phenylalanine-free protein supplements and low-phenylalanine foods), as assessed by the Investigator
- •Well-controlled phenylalanine levels, as assessed by a minimum of 75 percent of phenylalanine levels within the target recommended in each centre during the previous 3 months
- •Low phenylalanine diet started within the first 3 weeks of life
- •Parent(s) or guardian(s) willing to comply with all study procedures, maintain strict adherence with the diet, and willing and able to provide written, signed informed consent before any trial-related activities are carried out, as well as ability of child to comply with trial procedures
排除标准
- •Known hypersensitivity to Kuvan® or its excipients
- •Known hypersensitivity to other approved or non-approved formulations of BH4
- •Previous diagnosis of BH4 deficiency
- •Current use of methotrexate, trimethoprim or other dihydrofolate reductase inhibitors
- •Current use of medications that are known to affect nitric oxide synthesis, metabolism or action
- •Current use of experimental or unregistered drugs (other than sapropterin/BH4) that may affect the study outcomes or use of such agents within 30 days prior to Screening
- •Concurrent use of levodopa
- •Concurrent disease or condition that would induce repeatedly catabolic situations, or interfere with the trial participation, diet, or NC development, as assessed by the Investigator
- •Any condition that, in the view of the Investigator, renders the subject at high risk for failure to comply with treatment or to complete the trial
- •Participation in a clinical trial investigating any other agent than Kuvan® within the past 30 days
研究组 & 干预措施
Kuvan®
Experimental
干预措施: Kuvan® (Drug)
结局指标
主要结局
Mean Full Scale Intelligence Quotient (FSIQ) Score of the Wechsler Intelligence Scale for Children (WISC)-IV
时间窗: Year 7
次要结局
- Height compared to the World Health Organization (WHO) Growth Standards(up to 7 years)
- Weight compared to the World Health Organization (WHO) Growth Standards(up to 7 years)
- Blood levels of tyrosine, tryptophan, pre-albumin and methylmalonic acid(up to 7 years)
- Intelligence Quotient (IQ) score and sub-scores such as, verbal IQ, performance IQ, Full Scale Intelligence Quotient (FSIQ) of Wechsler Preschool and Primary Scale of Intelligence (WPPSI)-III(Baseline)
- Intelligence Quotient (IQ) score and sub-scores such as verbal comprehension, perceptual reasoning, working memory, and processing speed indexes and Full Scale Intelligence Quotient (FSIQ) of the Wechsler Intelligence Scale for Children (WISC)-IV(up to 7 years)
- Change from Baseline in FSIQ score at 2, 4 and 7 years(Baseline, and Year 2, 4 and 7)
- Dietary Phenylalanine tolerance(up to 7 years)
- Phenylalanine levels(up to 7 years)
- Index of Dietary Control (IDC)(up to 7 years)
- Percentage of tablets taken to assess treatment compliance(up to 7 years)
- Distribution of phenylalanine hydroxylase (PAH) genotype(Baseline)
- Number of subjects with Adverse Events and Serious Adverse Events(up to 7 years)
研究者
研究点 (6)
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