Phenylketonuria Nutritional, Metabolic, Endocrine Classical phenylketonuria
Conditions
Interventions
The study product (PKU GOLIKE PLUS 16+) is a phe-free protein substitute for the dietary management of patients with PKU, aged =16 years. Specifically, it is an amino acid mixture engineered with a mo
Sponsors
Applied Pharma Research (Switzerland)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female patients with a diagnosis of phenylketonuria, aged =16 years 2. Subjects who are already taking a protein substitute for PKU and are willing to replace at least part of their prescription with the new study product for 7 days 3. Willing and able to give a written informed consent
Exclusion criteria
Exclusion criteria: 1. Presence of serious concurrent illness 2. Presence of current illnesses that could interfere with the study (for instance, inflammatory status, etc) 3. Status of pregnancy or lactation, for females 4. Likelihood that the patient will not be compliant with the protocol requirements, in the judgement of the Lead Dietitian 5. Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study 6. Use of antibiotics in the week prior to study entry 7. Use of sapropterin 8. Patients aged less than 16 years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Product acceptability (perceptions about visual appearance, smell, taste, after-taste and mouth feeling/palatability; overall liking of the study product and patient’s potential interest at taking it on a long-term basis), assessed using an Acceptability Questionnaire at the end of the 7-day treatment 2. Gastrointestinal tolerance, assessed by collecting data about gastro-intestinal signs and symptoms (including abdominal discomfort/pain, diarrhoea, constipation, bloating or abdominal distension, nausea, vomiting, flatulence, regurgitation, burping) by means of a daily diary during the 7-day treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Compliance with the study product assessed by means of a diary where the actual number of intakes versus prescribed intakes are recorded on a daily basis during the 7-day treatment 2. Metabolic control, evaluated by measuring fasting blood phe levels by routine biochemical blood spot testing for phe at the beginning and at the end of the trial | — |
Countries
England, United Kingdom
Contacts
Public ContactGiorgio Reiner
Outcome results
None listed