Tetrahydrobiopterin (BH4) treatment in Phenylketonuria - comparing different practices of dosing regimen - BH4 dose

niversity Medical Center Groningen0 个研究点目标入组 28 人开始时间: 2021年11月24日最近更新: 2021年11月29日

适应症

试验速览

暂无简介。

•In order to be eligible to participate in this study, a subject must meet all of the following criteria:
•1. Diagnosed with phenylketonuria by newborn screening.
•2. Diagnosed as a BH4-responder after a 48-h BH4 loading test and a treatment trial
•3. Currently uses sapropterin as part of treatment (with a minimum of three months)
•4. Age = 4 years
•5. Good metabolic control for the previous 6 months, meaning 75% of the Phe levels are within therapeutic target levels with a minimum of four values.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 8
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 16
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•A potential subject who meets any of the following criteria will be excluded from participation in this study:
•Pregnancy, planning pregnancy or lactation

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