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

• Register: ClinicalTrials.gov
• Last refreshed on: 27 February 2024
• Main ID: NCT03771391
• Date of registration: 07/12/2018
• Prospective Registration: No
• Primary sponsor: Vitaflo International, Ltd
• Public title: A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU GMP In PKU
• Scientific 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.
• Date of first enrolment: December 6, 2018
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03771391
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Single (Participant).
• Phase: N/A

Countries of recruitment

Germany

Contacts

• Name: Peter Freisinger
• Affiliation: Kreiskliniken Reutlingen GmbH

Key inclusion & exclusion criteria

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



Age minimum: 10 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Phenylketonurias

Intervention(s)

Dietary Supplement: PKU Sphere

Primary Outcome(s)

Change in blood Phe [Time Frame: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.]

Secondary Outcome(s)

Dietary intake [Time Frame: Weeks 1 and 16.]

Gastrointestinal symptoms questionnaire [Time Frame: Weeks 1, 4, 8, 12 and 16]

Acceptance questionnaire [Time Frame: Weeks 1, 4, 8, 12 and 16]

Change in blood tyrosine [Time Frame: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.]

Satiety questionnaire [Time Frame: Weeks 1, 4, 8, 12 and 16]

Stool patterns [Time Frame: Weeks 1, 4, 8, 12 and 16]

Protein substitute intake [Time Frame: Weeks 1, 4, 8, 12 and 16]

Secondary ID(s)

F-2018-043

MCT-W-PKUSp-2017-06-29

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Johannes Gutenberg University Mainz

Kreiskliniken Reutlingen