Status:
COMPLETED
PANDA: PKU Amino Acid Evaluation
Lead Sponsor:
Nutricia Research
Conditions:
Phenylketonuria (PKU)
Eligibility:
All Genders
2-12 years
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 Pheny...
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 in...
Eligibility Criteria
Inclusion
- Both PKU and Non-PKU comparison subjects:
- Age ≥ 2 and ≤ 12 years
- Willing and able to provide informed consent by parents or legal representatives (and assent if required by local law/regulations)
- One subject per family
- PKU subjects specific inclusion criteria:
- PKU subjects identified by newborn screening and started low Phe diet before 1 month age
- Usage of at least two Phe-free protein substitutes on a daily basis for at least 26 consecutive weeks up to Visit 1
- 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:
- Same age (±3 years) and sex as an included PKU subject
Exclusion
- Both PKU and Non-PKU comparison subjects:
- Current psychiatric disorders
- Severe hepatic, thyroid or renal dysfunction
- Acute illnesses like fever, flu, diarrhea, or vomiting (subjects should be symptom free for a week prior to V1)
- Serious conditions (e.g. cancer, hydrocephalus, fatal heart disease)
- 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:
- Use of BH4, or drugs that may interfere with main outcomes
- Non-PKU comparison subjects specific exclusion criteria:
- PKU diagnosis or any other diagnosed disorder of amino or organic acid metabolism
Key Trial Info
Start Date :
September 10 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
April 14 2023
Estimated Enrollment :
37 Patients enrolled
Trial Details
Trial ID
NCT04086511
Start Date
September 10 2019
End Date
April 14 2023
Last Update
June 2 2023
Active Locations (2)
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1
Dr. P. Verloo
Ghent, Belgium
2
Birmingham Children's Hospital
Birmingham, United Kingdom