Hyperphenylalaninaemia, Phenylketonurias
Conditions
Brief summary
This study will evaluate the compliance, acceptability, gastrointestinal (GI) tolerance and safety of a lower calorie amino acid based liquid protein substitute for patients with Phenylketonuria (PKU) or hyperphenylalaninemia (HPA).
Interventions
DIETARY_SUPPLEMENTLow calorie protein substitute
The evaluated dietary supplement is a lower calorie amino acid based liquid protein substitute designed for patients with PKU.
Sponsors
Nutricia UK Ltd
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female * 3 years of age or above * Diagnosed with classical or variant type phenylketonuria (PKU), or hyperphenylalaninemia (HPA) * Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalent daily, for at least 1 month prior to study commencement * Have a prescribed daily Phe allowance * Written informed consent from patient, or from parent / carer if applicable
Exclusion criteria
* Pregnant or lactating * Requiring enteral tube or parenteral nutrition * Major hepatic or renal dysfunction * Participation in other studies within 1 month prior to entry to this study * Allergy to any of the study product ingredients * Investigator concern around the patient being underweight (e.g.: BMI lower than 18.5kg/m2 for adults) and/or having an eating disorder * Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Compliance | Daily for 31 days | Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Acceptability: Tick-box questionnaire | Recorded at baseline and end of intervetnion (day 31) | Tick-box questionnaire on overall liking and acceptability of product (ie dislike a lot, dislike moderately, neutral, like moderately, like a lot) |
| Blood phenylalanine and other amino acid levels | Recorded at baselien and end of intervention (day 31) | Blood spot test. |
| Gastro-intestinal tolerance | recorded at baseline, beginning of intervention and end of intervention(12 out of 31 days in total) | Questionnaire detailing any GI symptoms, severity (none, mild, moderate, severe) and change from usual. |
| Nutrient intake | Recorded at basleine and end of intervention (day 31) | 24h dietary recall. |
| Anthropometry | Recorded at baseline and end of intervention (day 31) | Measurements of height (m) and weight (kg) at baseline and end of study. |
Outcome results
None listed