Phenylketonuria
Conditions
Brief summary
This study will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU).
Interventions
DIETARY_SUPPLEMENTGlycomacropeptide-based protein substitute
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 * Over 3 years of age * Diagnosed with classical or variant type phenylketonuria * Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalents, for at least 1 month prior to trial commencement * Have a prescribed daily Phe allowance * Written informed consent from patient, or from parent / carer if applicable
Exclusion criteria
* Currently prescribed Sapropterin or similar tetrahydrobiopterin based medication * Pregnant or lactating * Requiring parenteral nutrition * Major hepatic or renal dysfunction * Participation in other studies within 1 month prior to entry of this study * Allergy to any of the study product ingredients, including milk protein or soya * Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Blood Phenylalanine (and other amino acids) | 3 points throughout 28 days | Blood spot test |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Product compliance questionaire | 31 days | Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount. |
| Product acceptability questionnaire | 2/31 days | Breif tick-box questionaire on overal liking and acceptability of product |
| Patient reproted nutrient intake (3 day food diaries) | 6/31 days | 3 day food diaries, subsequently analysed in dietary software. |
| Gastro-intestinal tolerance questionaire | 12/31 days | Questionaire detailing any GI symptoms, severity and change from usual |
| Antropometry (hieight and weight) | 2/31 days | Measurements of height and weight at baseline and end of study |
| Safety (Adverse events reporting) | 31 days | Reporting of adverse events throughout study |
| Pateint-reported appetite (visual analogue scale) | 6/31 days | Measures of overal appetite and satiety via visual analouge scale |
Countries
United Kingdom
Outcome results
None listed