Phenylketonuria (PKU) and HyperphenylalaninemiaBrain Diseases, Metabolic, InbornBrain Disorder, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Disease, InbornGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases
Investigational drug(s) / intervention(s)
Slow release, phenylalanine-free protein substitute tablets
Slow release, phenylalanine-free protein substitute tablets: Subjects who currently take a concentrated second stage phenylalanine-free protein substitute will be recruited. Subjects will take the study product for 7 days. Subjects will replace some or all of their usual protein substitute with the new product. The amount of study product prescribed will be calculated to provide the same amount of protein as their usual protein substitute.
Study summary
XPhePiccosMR is a prospective, single-arm, open label, observational study that aims to evaluate the acceptability and tolerability of XPhe Piccos, a slow-release, phenylalanine-free protein substitute in the form of tiny tablets, for use in the dietary management of phenylketonuria (PKU) in children aged three years and older.
Eligibility
Sex
ALL
Min age
3 Years
Max age
18 Years
Healthy volunteers
No
Inclusion Criteria:
* Diagnosis of PKU or HPA requiring a phenylalanine-free protein substitute.
* Subjects who are already taking a phenylalanine-free protein substitute and are willing to try the study product for 7 days.
* Subjects aged 3 years and above.
* Written informed assent/consent obtained from subject and/or parental caregiver, depending on subject age.
Exclusion Criteria:
* Presence of serious concurrent illness.
* Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
* Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
* Any subject having taken antibiotics over the previous 2 weeks leading up to the study.
* Subjects less than 3 years of age
Primary outcome measure(s)
Compliance — daily; from day 1 to day 7 Compliance with currently prescribed protein substitue will be assessed at baseline. Usage and compliance with the study product will subsequently assessed daily from days 1-7 using standardised questionnaires, where patients document the amount of consumed study product vs the prescribed doses.
Trial sites (1)
Facility
City
Region
Status
Birmingham Children´s Hospital
Birmingham
England
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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