N/A
N=16
Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
Phenylketonurias
Bottom Line
View on ClinicalTrials.gov: NCT05487378 ↗Enrolled (actual)
16
Serious AEs
0.0%
Results posted
Jun 2025
Primary outcome: Primary: Blood Phe After 7 Days of Each Treatment — 357.5; 294.0; 346.8; 442.4 µmol/L
Study Design & Population
- Study type
- Interventional
- Phase
- N/A
- Interventions
- PKU GOLIKE (Dietary_supplement)
- Age
- Pediatric · 5+ yrs
- Sex
- All
- Sponsor
- APR Applied Pharma Research s.a.
- Primary completion
- May 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Blood Phe After 7 Days of Each Treatment |
357.5; 294.0; 346.8; 442.4 | — |
| SECONDARY Dosage of Tyr in Blood (Umol/L) With Dried Blood Spots Before Breakfast |
46.4; 62.1; 49.5; 51.6 | — |
Summary
This is a 2 arm, randomised, controlled, cross-over study in 16 children with PKU. Subjects who are currently taking a Phe free/low Phe protein substitute will be recruited for a 31-day trial.
Patients will be randomised to receive:
1. The study product for 7 days as their last dose of protein substitute for the day (at least one sachet with 15g PE) in an amount equivalent to their usual protein substitute PE; or
2. An amino acid protein substitute for all daily doses for 7 days; followed by a 2-week washout period on their usual protein substitute,
and then 7 days of the other study arm.
During this time, patients/caregivers will be asked to:
* Collect 3 finger prick blood spots on days -1, 0, 6, 7, 20, 21, 27 and 28.
* Collect urine sample, second void of the day on days 0, 7, 21 and 28.
* Complete a questionnaire on sleep quality on day 0, 7, 21 and 28.
* Complete a 24 hour food diary on days -1, 0, 6, 7, 20, 21, 27 and 28.
APR will supply the study product for participants free of charge.
Eligibility Criteria
Inclusion Criteria
- Male and female PKU patients ≥5 years and ≤16 years of age.
- Patients diagnosed with PKU via new born screening.
- Taking a Phe free/low Phe protein substitute
- On a low phenylalanine diet .
- Absence of neurological deficiencies.
- Adherence with dietary management and protein substitute.
- Able to understand and comply with the requirements of the investigation and sign the Informed Consent Form/Assent form.
Exclusion Criteria
- Age 16 years old.
- Patients with mild PKU or HPA.
- On sapropterin therapy.
- Patients with late diagnosis of PKU and neurological problems.
- History of hypersensitivity to any excipients/components of the investigational product.
- Pregnancy or breastfeeding during the study.
- Any moderate to severe acute illness which in the opinion of the Investigator would interfere with the study procedures or study outcome.
- History of poor co-operation, non-adherence with dietary management, or poor adherence to investigation procedures.
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
Data sourced from ClinicalTrials.gov (NCT05487378). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.