PANDA: PKU Amino Acid Evaluation

Phenylketonuria (PKU) Clinical Trial

— PANDA  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04086511
• Other study ID #: MBB18TA18444
• Status: Completed
• Start date: September 10, 2019
• Est. completion date: April 14, 2023

Study information

• Verified date: September 2021
• Source: Nutricia Research
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 37
• Est. completion date: April 14, 2023
• Est. primary completion date: December 21, 2022
• Gender: All
• Age group: 2 Years to 12 Years

Eligibility

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 exclusion criteria:

6. Use of BH4, or drugs that may interfere with main outcomes

Non-PKU comparison subjects specific exclusion criteria:

7. PKU diagnosis or any other diagnosed disorder of amino or organic acid metabolism

Related Conditions & MeSH terms

• Phenylketonuria (PKU)
• Phenylketonurias

Locations

Country	Name	City	State
Belgium	Dr. P. Verloo	Gent
United Kingdom	Birmingham Children's Hospital	Birmingham

Sponsors (1)

Lead Sponsor	Collaborator
Nutricia Research

Countries where clinical trial is conducted

Belgium,  United Kingdom, 

Outcome

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