Phenylketonuria Nutritional, Metabolic, Endocrine Phenylketonuria
Conditions
Interventions
The study product (PKU GOLIKE PLUS 3-16) is a phenylalanine-free protein substitute for the dietary management of patients with PKU, aged 3-16 years. Specifically, it is an amino acid mixture engineer
Sponsors
APR Applied Pharma Research SA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female patients with a diagnosis of phenylketonuria, aged 3-16 years 2. Subjects who are already taking a protein substitute for PKU and are willing to try the study product for 7 days 3. Written informed consent obtained from parental caregiver
Exclusion criteria
Exclusion criteria: 1. Presence of serious concurrent illness 2. Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements 3. Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study 4. Any children having taken antibiotics over the previous 2 weeks leading up to the study 5. Children aged less than 3 years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Product acceptability (ease of use and palatability, i.e. perceptions about taste, appearance, smell) assessed using a questionnaire at day 7, at the end of the treatment 2. Product tolerability assessed using a questionnaire collecting data about gastro-intestinal side effects (i.e. diarrhoea, constipation, nausea, vomiting, abdominal pain) on a daily basis during the 7-day treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Compliance with the study product assessed using standardized questionnaires 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 phenylalanine levels by routine biochemical blood spot testing for phenylalanine and tyrosine at the beginning and at the end of the trial | — |
Countries
United Kingdom
Outcome results
None listed