CompletedNot Applicable

Evaluation of PKU Sphere in Italy

Italy13 participantsStarted 2020-08-10

Plain-language summary

20 participants with PKU will build-up their dietary intake of PKU sphere over 2-16 weeks, depending on their level of metabolic control whilst doing so. Participants will complete a gastrointestinal specific and PKU specific questionnaire at the Baseline clinic visit and record the amount of PKU sphere taken each day. Dried blood spots are taken twice per week. Once built up to a clinically appropriate intake of PKU sphere, or after 16 weeks, participants enter a 4-week Evaluation Period. The amount of PKU sphere taken per day continues to be recorded by participants. Gastrointestinal tolerance over the preceding seven days is recorded at the end of each week. Evaluations of PKU sphere's palatability are made at the end of weeks two and four of the Evaluation Period. Dried blood spots are taken once per week. The participant attends an End of Study Visit at the clinic and the investigator decides whether they should continue taking PKU sphere.

Who can participate

Age range

3 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: * Patients diagnosed with PKU or hyperphenylalaninemia via new born screening and early treated, requiring \>50% of protein intake from protein substitute. * Aged 3 years and above. * Female patients of childbearing potential must have a negative urine pregnancy test prior to completing the screening procedures. * Willingly given, written, informed consent from the participant, 18 or more years, or from the parent/guardian for participants \< 18 years. * Willingly given, written assent (if appropriate) for those \< 18 years. Exclusion Criteria: * Pregnant, planning pregnancy or breastfeeding. * Intake of sapropterin dihydrochloride (Kuvan), PEG PAL or large neutral amino acids within 30 days prior to screening visit. * Patients currently taking their maximum recommended amount of protein substitute from a GMP based protein substitute. * Patients who are unable/unwilling to replace their current GMP protein substitute (if any) with PKU sphere. * Patients with known soya, milk or fish allergies / intolerance. * Individuals, who in the opinion of the investigator, are unable to comply with the requirements of the protocol. * Any co-morbidity, which in the opinion of the Investigator, would preclude participation in the study. * Patients who are currently participating in, plan to participate in, or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to screening visit.

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

Change in adherence to recomended amounts of PKU sphere

Timeframe: Recorded daily from start of 2-16 week Build-up and Stabilisation Period to end of 4 week Evaluation Period, assessed by HCPs periodically as per routine practice.

Change in gastrointestinal tolerance

Timeframe: Data on previous 7 days collected by patients at the end of each Evaluation Period week. This data will be assessed by HCPs periodically as per routine practice.

Patient evaluation of PKU sphere's palatability, Evaluation Period week 2

Timeframe: End of Evaluation Period week 2.

Patient evaluation of PKU sphere's palatability, Evaluation Period week 4

Timeframe: End of Evaluation Period week 4.

Change in metabolic control (phenylalanine) over Build-up and Stabilisation Period.

Timeframe: Dried blood spots taken twice weekly during 2-16 week Build-up and Stabilisation Period.

Change in metabolic control (phenylalanine) over Evaluation Period.

Timeframe: Dried blood spots taken once weekly during 4-week Evaluation Period.

Trial details

NCT IDNCT04679467

SponsorVitaflo International, Ltd

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-07-31

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