EUCTR2009-015844-41-DE进行中（未招募）1 期
A Phase IV 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. - KOGNITO (Kuvan®’s effect on the cOGNITion of children with phenylketOnuria)
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 60
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
入选标准
- •The target population for this trial is children with confirmed diagnosis of PKU.
- •Male or female outpatients, 4 to 5 years of age (=4 and <6) at the time of ICF signature by parent(s) or guardian(s).
- •Confirmed clinical and biochemical diagnosis of PKU, including at least two separate blood Phe levels =400 µmol/L.
- •Defined pre-Kuvan®/-tetrahydrobiopterin (BH4) dietary Phe 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 Phe levels and/or Phe tolerance.
- •- For subjects not treated with Kuvan®/BH4 at Screening: a response test has been performed during Screening or is available from the patient’s medical records and satisfies the 3 following criteria: a decrease in blood Phe levels of at least 30% was observed after at least 24 hours with a dose of at least 10 mg/kg/day.
- •IQ =70, as assessed with the WPPSI™-III 2nd part.
- •Note: If a subject has undertaken the same test up to 3 months before Screening as part of his/her clinical follow-up, the score can be recorded and the test doesn’t need to be repeated.
- •Good adherence with dietary treatment (including prescribed dietary Phe restriction and prescribed amounts of Phe-free protein supplements and low-Phe foods), as assessed by the Investigator.
- •Well-controlled Phe levels, as assessed by a minimum of 75% of Phe levels within the target recommended in each centre during the previous 3 months.
- •Low Phe 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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 30
- •F.1.2 Adults (18-64 years) no
- •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
- •Known hypersensitivity to Kuvan® or its excipients.
- •Known hypersensitivity to other approved or non-approved formulations of tetrahydrobiopterin.
- •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.
研究者
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