A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU

Phenylketonurias Clinical Trial

— GMP In PKU  

Official title:

Multicentre Study Project to Evaluate the New Generation of Protein Supplements With Glycomacropeptide (GMP) in Patients With Phenylketonuria (PKU) Aged 10 Years and Older Over a 16 Week Period.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03771391
• Other study ID #: MCT-W-PKUSp-2017-06-29
• Secondary ID: F-2018-043
• Status: Completed
• Phase: N/A
• Start date: December 6, 2018
• Est. completion date: July 31, 2023

Study information

• Verified date: February 2024
• Source: Vitaflo International, Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study aims to measure changes in participants' blood phenylalanine (Phe) levels in participants with PKU when switching from a conventional amino acid based protein substitute to a GMP based protein substitute (PKU Sphere) over a 16 week period.

Description:

This is a stepped wedge, open-label study of PKU Sphere for the dietary management of participants with Phenylketonuria. It will be carried out with 33 participants who will be randomised into one of three clusters. The total study length per patient is 16 weeks. Each cluster transitions from taking their standard amino acid product alone to incorporating PKU Sphere (at least 50% of their protein substitue requirement) at different timepoints: week 5, 9 or 13.

Participants will have a face-to-face clinical review at the time of recruitment (week 0) and at 16 weeks (completion of the study). They will also have an additional telephone review when they switch onto PKU Sphere.

Blood samples will be taken at baseline, and then every two weeks for the duration of the study.

In addition to these study visits and procedures, routine clinical care will continue in line with local guidelines.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: July 31, 2023
• Est. primary completion date: October 30, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years and older

Eligibility

Inclusion Criteria:

• Diagnosis of PKU made in neonatal period

• Treated by a low-phenylalanine diet and necessity of intake of at least one protein substitute per day

• Aged 10 years and above

• Three blood Phe measurements taken within the preceding six months

• Willing to replace current amino acid based protein substitute with PKU Sphere

• Ability to take at least 50% of protein requirements as PKU Sphere

• Ability to comply with the study protocol, in the opinion of the investigator

• Willingly given, written, informed consent from patient or parent/guardian

• Willingly given, written assent (if appropriate)

Exclusion Criteria:

• Women who are pregnant or planning to become pregnant during the study period

• Participants with atypical PKU (e.g. BH4 deficient)

• Intake of sapropterin dihydrochloride (Kuvan) in the six weeks prior to recruitment in the study

• Patients with soya, milk or fish allergies

• Any other severe disease

Related Conditions & MeSH terms

• Phenylketonurias

Intervention

Dietary Supplement:
• PKU Sphere
PKU sphere is a powdered, low phenylalanine protein substitute containing a balanced mix of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, vitamins, minerals and docosahexaenoic acid (DHA). With sugar and sweetener.

Locations

Country	Name	City	State
Germany	Kreiskliniken Reutlingen	Reutlingen	Baden-Württemberg

Sponsors (3)

Lead Sponsor	Collaborator
Vitaflo International, Ltd	Johannes Gutenberg University Mainz, Kreiskliniken Reutlingen

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in blood Phe	Change in blood Phe concentrations when switching from an amino acid based protein substitute to a GMP based one (PKU Sphere). A minimum of five (5) blood samples (including a baseline measurement and one from each treatment arm) will be required for a patient to be included in the final analysis.	Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
Secondary	Change in blood tyrosine	Impact of a GMP based protein substitute on blood tyrosine levels. A minimum of five (5) blood samples (including a baseline measurement and one from each treatment arm) will be required for a patient to be included in the final analysis.	Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
Secondary	Dietary intake	Compliance with a low protein diet - 3-day food diary.	Weeks 1 and 16.
Secondary	Protein substitute intake	Compliance with the recommended volume of protein substitute - 3-day protein substitute diary.	Weeks 1, 4, 8, 12 and 16
Secondary	Stool patterns	3-day diary noting stool type (Bristol stool chart).	Weeks 1, 4, 8, 12 and 16
Secondary	Satiety questionnaire	Short questionnaire asking participants to consider their satiety levels over the last 7 days, asking them if it has increased, stayed the same or decreased. Questionnaire validated as part of a bachelor thesis.	Weeks 1, 4, 8, 12 and 16
Secondary	Acceptance questionnaire	Questionnaire to collect information about taste, odour and practicability of the protein substitutes. The questions are answered on a scale of 1 (bad) to 5 or 6 (good). Questionnaire validated as part of a bachelor thesis.	Weeks 1, 4, 8, 12 and 16
Secondary	Gastrointestinal symptoms questionnaire	Short questionnaire asking participants to consider their general gastrointestinal health over the previous 7 days. The occurrence of various GI symptoms are given as never, rare, often or always. Questionnaire validated as part of a bachelor thesis.	Weeks 1, 4, 8, 12 and 16