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Could a PKU diet reshape the gut microbiome? a study investigates

NCT ID NCT07728032

Knowledge-focused Sponsor: Birmingham Women's and Children's NHS Foundation Trust Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 27, 2026 · Last updated Jul 28, 2026 · Updated 1 time

Summary

This observational study examines how a phenylalanine-restricted diet and PKU medications like sapropterin influence the gut microbiome in children aged 3-17. Researchers will compare stool samples from children with PKU and their healthy siblings to see if diet or drugs alter gut bacteria and metabolites. The goal is to better understand the link between PKU treatment, diet, and gastrointestinal health.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Phenylalanine-restricted diet and sapropterin dihydrochloride (Kuvan)
What this could lead to
If successful, this research could reveal how diet and medication shape the gut microbiome in PKU, potentially leading to better nutritional strategies for children with the condition.
What could go wrong
This is an observational study, not a treatment trial, so it cannot prove cause and effect. The findings may not apply to all children with PKU.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 148 people

The number the study aims to enrol. It can still change while the study runs.

Started

Oct 2025

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Children and adolescents aged 3-17 years with early-treated phenylketonuria receiving clinical care at Birmingham Children's Hospital are recruited into one of three cohorts according to their current treatment: a phenylalanine-restricted diet alone, sapropterin, or sepiapterin. One healthy household control aged 3 years or older, without phenylketonuria or another inherited metabolic disorder, is recruited for each participant with phenylketonuria. All participants are enrolled according to predefined eligibility criteria.

Ages

3 to 17 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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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Conditions

The condition(s) this trial relates to.

phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Phenylketonuria

How to take part

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  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

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Contacts and locations

Locations

  • Birmingham Children's Hospital

    RECRUITING

    Birmingham, Birmingham, B4 6NH, United Kingdom

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