Evaluation of PKU Explore France

Launched by VITAFLO INTERNATIONAL, LTD · May 18, 2021

Keywords

ClinConnect Summary

The PKU Explore France trial is a study designed to see how well a special food product, called PKU Explore, works for infants aged 6 months to 3 years with phenylketonuria (PKU). PKU is a condition that makes it hard for the body to break down a protein found in many foods, so children with PKU need to follow a strict low-protein diet. The study will look at how easy it is for families to use this product, how well the children tolerate it, and how it affects their growth and blood levels of phenylalanine, which is the substance they need to limit in their diet.

To be eligible for the trial, children must have a confirmed diagnosis of PKU and already be on a low-protein diet, using a spoonable protein substitute. They should be well-managed, meaning their phenylalanine levels are stable. Parents or guardians must be able to give consent for their child to participate. Throughout the study, families can expect to monitor how their child reacts to the PKU Explore product and share their experiences. It's important to note that children with certain other health issues or allergies cannot participate, and the study is currently active but not recruiting new participants.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free L-amino acid protein substitute.
• • Aged between 6 months and 3 years (inclusive at screening).
• • Already taking part of their protein substitute in a spoonable form OR is at the stage in their PKU management when a second stage spoonable protein substitute is recommended to commence.
• • Well-controlled PKU, evidenced by the latest three routine blood spots being within the acceptable range, in the investigator's opinion.
• • Able to comply with the study protocol and take the study product, according to the opinion of the investigator.
• • Willingly given, written, informed consent from parents/guardian.
• Exclusion Criteria:
• • Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and Phe-free L-amino acid supplements.
• • Diagnosis of a concurrent condition which may adversely affect developmental progression and feeding ability.
• • Known milk or fish allergy/intolerance.
• • 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 the screening visit.
• • Existing significant GI issues which may affect compliance with the study protocol, according to the opinion of the investigator.
• • Any medical conditions precluding the study intervention, which in the opinion of the investigator may impact on metabolic control during the study period.
• • Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator (must be recorded in patient case record file).
• • Where applicable, patients NOT covered by Health Insurance System and/or not in compliance with the recommendations of National Law in force.