Market Research - Acceptability Study for a New PKU Protein Substitute

Not Applicable

Completed

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

• Registration Number: NCT04318509
• 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: 2019-02-04
• Primary Completion: 2019-05-19
• Study Completion: 2019-05-19
• Status Verified: 2020-03
• Study First Submitted: 2020-03-20
• Study First Submitted QC: 2020-03-20
• Last Update Submitted: 2020-03-20
• Study First Posted: 2020-03-24
• Last Update Posted: 2020-03-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

Inclusion Criteria

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

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 GMPOWER	GMPOWER	(casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years

Primary Outcome Measures

Name	Time	Method
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.
Product compliance	7 days	Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
Product palatability	7 days	Questionnaire data captured to evaluate taste
Product acceptability	7 days	Brief tick-box questionnaire on overall liking and acceptability of product

Trial Locations

Locations (2)

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

Bristol Royal Hospital for Children; 🇬🇧 Bristol, United Kingdom