Market Research - Acceptability Study for a New PKU Protein Substitute

Metabolic Disease Clinical Trial

Official title:

Market Research - Acceptability Study for a New PKU Glycomacropeptide Based Protein Substitute (Dr Schär- Mevalia Amino Acids) Mevalia PKU GMPower - Vanilla

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04318509
• Other study ID #: GMPower1
• Status: Completed
• Phase: N/A
• Start date: February 4, 2019
• Est. completion date: May 19, 2019

Study information

• Verified date: March 2020
• Source: Dr. Schär AG / SPA
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: May 19, 2019
• Est. primary completion date: May 19, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 16 Years

Eligibility

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.

Related Conditions & MeSH terms

• Inborn Errors of Metabolism
• Metabolic Disease
• Metabolic Diseases
• Metabolism, Inborn Errors
• Phenylketonurias
• PKU

Intervention

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

Locations

Country	Name	City	State
United Kingdom	Birmingham Children's Hospital	Birmingham
United Kingdom	Bristol Royal Hospital for Children	Bristol

Sponsors (1)

Lead Sponsor	Collaborator
Dr. Schär AG / SPA

Country where clinical trial is conducted

United Kingdom, 

Outcome

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