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Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU

Recruitingn/aAges 3 Years - 17 YearsAllBirmingham Women's and Children's NHS Foundation …
NCT07728032 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

Phenylketonuria (PKU) is an inherited disorder of phenylalanine (Phe) metabolism. The mainstay of treatment is a Phe-restricted diet, which aims to maintain blood Phe concentrations within the recommended range and prevent neurological complications. Some individuals with PKU respond to pharmacological treatments, including sapropterin, a synthetic form of tetrahydrobiopterin (BH4), or sepiapterin. These treatments may increase Phe tolerance and allow a less restrictive diet. Diet is an important determinant of gut microbiota composition and function. However, the effects of the Phe-restricted diet and pharmacologically enabled dietary relaxation on the gut microbiota in PKU remain poorly understood. This observational study includes children and adolescents with PKU aged 3-17 years attending Birmingham Children's Hospital. Participants include those managed exclusively with a Phe-rest

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📋 Eligibility rules (as published)
Inclusion Criteria: Participants with PKU: * Aged 3-17 years. * Confirmed diagnosis of phenylketonuria following newborn screening. * Receiving ongoing clinical management for PKU. * For the diet-only cohort: managed with standard phenylalanine-restricted dietary treatment and not receiving sapropterin or sepiapterin. * For the sapropterin cohort: receiving sapropterin for at least three consecutive months and having achieved at least a 100% increase in natural protein tolerance compared with the pre-treatment prescription. * For the sepiapterin cohort: receiving sepiapterin for at least three consecutive months and having achieved at least a 100% increase in natural protein tolerance compared with the pre-treatment prescription. Healthy household controls: * Aged 3 years or older. * Living in the same household as a participating child or adolescent with PKU. * No known diagnosis of PKU or another inherited metabolic disorder. Exclusion Criteria: * Congenital malformations. * Chronic gastrointestinal disease. * Endocrine, liver, or kidney disease. * Other chronic medical conditions likely to affect gut microbiota composition. * Following a therapeutic diet for a medical condition other than PKU within the six months before stool sample collection.
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What it tests

  • Phenylalanine-restricted diet alone
  • Sapropterin Dihydrochloride
  • Sepiapterin
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Where

1 site
Birmingham Children's Hospital
Birmingham, United Kingdom
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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