Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute

Not Applicable

Completed

• Conditions: Phenylketonurias; PKU; Inborn Errors of Metabolism; Metabolic Disease
• Interventions: Dietary Supplement: Motion

• Registration Number: NCT04309331
• Lead Sponsor: Dr. Schär AG / SPA

Brief Summary

The aim of this study is to demonstrate that a new protein substitute is acceptable and well tolerated in children with PKU.

Detailed Description

Not available

Study Timeline

• Study Start: 2018-07-01
• Primary Completion: 2018-09-19
• Study Completion: 2018-09-19
• Study First Submitted: 2020-02-13
• Status Verified: 2020-03
• Study First Submitted QC: 2020-03-12
• Last Update Submitted: 2020-03-12
• Study First Posted: 2020-03-16
• Last Update Posted: 2020-03-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• Diagnosis of PKU or PKU variant requiring a phenylalanine-free protein substitute
• Subjects who are already taking a phenylalanine-free protein substitute and are willing to try the study product for 7 days
• Patients aged 3 years of age to adulthood
• Written informed consent obtained from parental caregiver

Exclusion Criteria

• Presence of serious concurrent illness
• Investigator's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
• Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
• Any patients having taken antibiotics over the previous 2 weeks leading up to the study.
• Patients less than 3 years of age.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
PKU Motion	Motion	amino acid based protein substitute

Primary Outcome Measures

Name	Time	Method
Product palatability	7 days	Questionnaire data captured to evaluate taste
Product compliance	7 days	Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
Product acceptability	7 days	Brief tick-box questionnaire on overall liking and acceptability of product
Gastrointestinal tolerance	7 days	Questionnaire detailing any GI symptoms, severity and change from usual; • The collection of daily data about the gastro-intestinal tolerance of the protein substitute.

Trial Locations

Locations (1)

Birmingham Children's Hospital; 🇬🇧 Birmingham, United Kingdom