MedPath

Evaluation of PKU Explore France

Not Applicable
Active, not recruiting
Conditions
Phenylketonurias
Registration Number
NCT04898829
Lead Sponsor
Vitaflo International, Ltd
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Active, not recruiting
Sex
All
Target Recruitment
Not specified
Inclusion Criteria

Inclusion Criteria:<br><br> - A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free<br> L-amino acid protein substitute.<br><br> - Aged between 6 months and 3 years (inclusive at screening).<br><br> - Already taking part of their protein substitute in a spoonable form OR is at the<br> stage in their PKU management when a second stage spoonable protein substitute is<br> recommended to commence.<br><br> - Well-controlled PKU, evidenced by the latest three routine blood spots being within<br> the acceptable range, in the investigator's opinion.<br><br> - Able to comply with the study protocol and take the study product, according to the<br> opinion of the investigator.<br><br> - Willingly given, written, informed consent from parents/guardian.<br><br>Exclusion Criteria:<br><br> - Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary<br> intervention with a low protein diet and Phe-free L-amino acid supplements.<br><br> - Diagnosis of a concurrent condition which may adversely affect developmental<br> progression and feeding ability.<br><br> - Known milk or fish allergy/intolerance.<br><br> - Patients who are currently participating in, plan to participate in or have<br> participated in an interventional investigational drug, food or medical device trial<br> within 30 days prior to the screening visit.<br><br> - Existing significant GI issues which may affect compliance with the study protocol,<br> according to the opinion of the investigator.<br><br> - Any medical conditions precluding the study intervention, which in the opinion of<br> the investigator may impact on metabolic control during the study period.<br><br> - Use of additional macro/micronutrient supplements during the study period, unless<br> clinically indicated and prescribed by the investigator (must be recorded in patient<br> case record file).<br><br> - Where applicable, patients NOT covered by Health Insurance System and/or not in<br> compliance with the recommendations of National Law in force.

Exclusion Criteria

Not provided

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Adherence to the recommended amount of study product;Product palatability rated on a Likert scale by the patient after 28 days;Gastrointestinal tolerance daily diary as reported by the patient;Change in Phe levels
Secondary Outcome Measures
NameTimeMethod
Evaluation of growth for a maximum of two years follow up period;Product adherence for a maximum of two years follow up period by measuring the quantity of product intake;Product adherence for a maximum of two years follow up period by measuring the Phe levels
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