CompletedNot Applicable

Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia

United Kingdom14 participantsStarted 2017-05-01

Plain-language summary

This study centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. Fifty eligible adults (≥ 16 years) with proven phenylketonuria or hyperphenylalaninemia will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Following a 3-day baseline period, and in addition to routine nutritional management, patients will receive either one sachet of the new protein substitute daily (intervention) or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.

Who can participate

Age range

16 Years – 100 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male or female * Over 16 years of age * Diagnosed with proven PKU or hyperphenylalaninemia with an increased phenylalanine-tolerance/intake * Currently taking a maximum of 1 protein substitute per day (equal to 20g protein equivalent) * Have a minimum blood phenylalanine level of ≥ 600 umol/L (for PKU patients) * Have relaxed (if not stopped) their dietary and protein substitute regimen for at least 1 month prior to trial commencement * Have Written informed consent from patient Exclusion Criteria: * Pregnant or lactating * Requiring nutritional support (including enteral and 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 to comply with protocol requirements

Questions worth asking your doctor

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.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

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.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Nutritional Status (objective measure)

Timeframe: 2 days

Trial details

NCT IDNCT03167697

SponsorNutricia UK Ltd

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-09-20

View on ClinicalTrials.gov More Phenylketonurias trials