Evaluation of PKU Explore

• Register: ClinicalTrials.gov
• Last refreshed on: 27 February 2024
• Main ID: NCT03168399
• Date of registration: 24/05/2017
• Prospective Registration: Yes
• Primary sponsor: Vitaflo International, Ltd
• Public title: Evaluation of PKU Explore PKU Explore
• Scientific title: A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.
• Date of first enrolment: June 8, 2017
• Target sample size: 22
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03168399
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: N/A

Countries of recruitment

United Kingdom

Contacts

• Name: Anita MacDonald
• Affiliation: Birmingham Women's and Children's NHS Foundation Trust

Key inclusion & exclusion criteria

Inclusion Criteria:

i. A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free
L-amino acid supplements.

ii. Aged between 6 months and 5 years.

iii. Already taking a second stage concentrated protein substitute as part of their PKU
management OR is at the stage in their PKU management when a second stage concentrated
protein substitute is recommended to commence.

iv. Willingly given, written, informed consent from parent/guardian.

v. Willingly given, written assent (if appropriate).

Exclusion Criteria:

i. Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary
treatment with a low protein diet and Phe-free L-amino acid supplements.

ii. Diagnosis of a concurrent condition which may adversely affect developmental
progression and feeding ability.

iii. Participation in any other clinical trial/acceptability study.

iv. Any serious medical precluding the study intervention.

v. 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).



Age minimum: 6 Months
Age maximum: 5 Years
Gender: All

Health Condition(s) or Problem(s) studied

Phenylketonuria

Inborn Errors of Metabolism

Intervention(s)

Dietary Supplement: PKU Explore

Primary Outcome(s)

Ease of use questionnaire [Time Frame: Day 29]

Product compliance daily diary [Time Frame: Days 1-28]

Weekly phenylalanine control [Time Frame: Days 1-28]

GI tolerance daily diary assessing change [Time Frame: Days 1-7 and 22-28]

Secondary ID(s)

MCT-PKUE-2016-12-05

219119

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Birmingham Women's and Children's NHS Foundation Trust