A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The following parameters will be assessed: adherence to prescribed dietary intakes, palatability, usability, gastrointestinal tolerance, clinically relevant routine biochemical parameters, timeframe to transition and contribution of the express plus range to overall protein substitute intake over a 28 day period.
Age range
3 Years
Sex
ALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Questionnaire of self-reported adherence to the prescribed amount of study product
Timeframe: Days 1-28
Transition time
Timeframe: Transition period Weeks 1-6 (maximum)
Change in gastrointestinal tolerance from week 1 to week 4
Timeframe: Days 1-7 and days 21-28
Product acceptability rated on a Likert scale by the patient after 28-day intake
Timeframe: Visit 2 (end of 28 day evaluation period)
Change in general neophobia
Timeframe: Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)
Change in food neophobia
Timeframe: Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)
Nutritional suitability: change Phenylalanine levels
Timeframe: Visit 1 until visit 2 (day 28)
Nutritional suitability: change Tyrosine levels
Timeframe: Visit 1 until visit 2 (day 28)