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PANDA: PKU Amino Acid Evaluation

PANDA: A Cross-sectional Study to Measure Blood Amino Acid Levels in PKU Children on a Protein Substitute

NCT04086511•Nutricia Research

Summary

Phenylketonuria (PKU) is a rare inherited metabolic disorder, where subjects are born with a genetic deficiency in the phenylalanine hydroxylase enzyme (PAH), which leaves them unable to convert Phenylalanine (Phe) into Tyrosine (Tyr). PKU patients have specific dietary needs and must follow a restrictive diet in the aim of preventing toxic levels of the amino acid phenylalanine (Phe) accumulation.

Detailed Description

The exploratory study's main objective is to measure blood amino acid levels and to gain quantitative insights in children with PKU on a protein substitute with respect to evaluation of nutritional intake.

Eligibility

Age

2 - 12 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Both PKU and Non-PKU comparison subjects:
•1. Age ≥ 2 and ≤ 12 years
•2. Willing and able to provide informed consent by parents or legal representatives (and assent if required by local law/regulations)
•3. One subject per family
•4. PKU subjects identified by newborn screening and started low Phe diet before 1 month age
•5. Usage of at least two Phe-free protein substitutes on a daily basis for at least 26 consecutive weeks up to Visit 1
•6. Average Phe-level ≤360 µmol/L based on at least two blood Phe values from the past 12 months up to Visit 1
•7. Same age (±3 years) and sex as an included PKU subject

Exclusion Criteria

•Both PKU and Non-PKU comparison subjects:
•1. Current psychiatric disorders
•2. Severe hepatic, thyroid or renal dysfunction
•3. Acute illnesses like fever, flu, diarrhea, or vomiting (subjects should be symptom free for a week prior to V1)
•4. Serious conditions (e.g. cancer, hydrocephalus, fatal heart disease)
•5. Participation in any other clinical intervention studies involving test products concomitantly or within six weeks prior to entry into the study
•6. Use of BH4, or drugs that may interfere with main outcomes
•7. PKU diagnosis or any other diagnosed disorder of amino or organic acid metabolism

Locations (2)

Dr. P. Verloo

Ghent, Belgium

Birmingham Children's Hospital

Birmingham, United Kingdom

Key Dates

Start Date

September 10, 2019

Primary Completion Date

December 21, 2022

Completion Date

April 14, 2023

Last Updated

June 2, 2023

Enrollment

ACTUAL participants

Conditions

Phenylketonuria (PKU)

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 2, 2023Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

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PANDA: PKU Amino Acid Evaluation

Inclusion Criteria

Exclusion Criteria

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

AAV Gene Therapy Study for Subjects with PKU

A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With Phenylketonuria

Iron Supplements Effect on B-Phe Levels in PKU Patients During 3 Years Study

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU