PANDA: PKU Amino Acid Evaluation

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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04086511

Acronym

PANDA

Enrollment

Registered

2019-09-11

Start date

2019-09-10

Completion date

2023-04-14

Last updated

2023-06-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Phenylketonuria (PKU)

Brief 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.

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

CROSS_SECTIONAL

Eligibility

Sex/Gender

ALL

Age

2 Years to 12 Years

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 PKU subjects specific inclusion criteria: 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 Non-PKU comparison subjects specific inclusion criteria: 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 PKU specific

Design outcomes

Primary

Measure	Time frame	Description
Amino acid levels in blood	day 1	Measuring amino acid levels in blood \[μmol/L\]
Nutrient Intake	day 1 - day 3	Measured by three-day diet diary. Nutrients in \[mg/day\]

Countries

Belgium, United Kingdom

Outcome results