Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria

What Was Tested

In this phase 4 trial, researchers set out to test L-Carnitine.

Effect of L-carnitine supplementation on phenylalanine and brain-derived neurotrophic factor levels in infants and children with phenylketonuria L-carnitine is mainly obtained from foods such as red meat and milk that are limited for PKU patients due to dietary protein restriction.

So, L-carnitine deficiency has been described in phenylketonuric patients The aim of this study is to : 1.

Compare level of Brain-derived neurotrophic factor in infants and children with phenylketonuria and healthy infants and children. 2.

It had two arms (groups): 88 patients diagnosed as case of phenylketonuria (experimental) and 88 control healthy children same age and sex (no intervention).

The registry classes the interventions as drug.

More on the Plan

Any patient with other metabolic, genetic or neurological diseases. 2.

Statistical analysis: The collected data will be coded, processed and analyzed using SPSS program (Version 29) .

The appropriate statistical tests will be used when needed.

Ethical consideration: * Study protocol will be submitted for approval by IRB. * Informed written consent will be obtained from each participant sharing in the study. * Confidentiality and personal privacy will be respected in all levels of the study.

Who Took Part

Enrollment: 176 actual participants took part.

It was open to people of any sex aged from 1 Month up to 18 years.

Healthy volunteers could join, not only people with the condition.

Examples of who could join: Male and female PKU patients diagnosed and following up at Genetics and Metabolic Outpatient Clinic. Age from one month up to 18 years.

The full record lists 2 inclusion and 2 exclusion criteria; among the exclusions: any patient with other metabolic, genetic or neurological diseases; patients who and/or their guardians refused to sign the consent.

How the Study Was Run

It was run as a randomized, sequential and open-label study.

Random assignment meant chance, not the researchers, decided who got L-Carnitine and who got the comparison, which limits bias.

Being open-label, the L-Carnitine study did not mask who received what.

It started in 2025-02-01 with completion listed for 2026-03-01.

It ran at 1 site in Egypt.

Its stated primary purpose is supportive care.

What This Means in Plain Language

Put simply, this is a phase 4, randomized trial of L-Carnitine in 176 people.

Its status is active not recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the L-Carnitine compound page, all human trials.