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

NCT03771391 | Completed

• 2 other identifiers: MCT-W-PKUSp-2017-06-29F-2018-043
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Phenylketonurias

Keywords

Phenylketonuria; PKU; Child; Children; Adult; Adults; GMP; Glycomacropeptide; Amino acid mixture; Amino acid; Protein supplement; Protein substitute

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (7)

• Change in blood tyrosine

  Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.

• Dietary intake

  Weeks 1 and 16.

• Protein substitute intake

  Weeks 1, 4, 8, 12 and 16

• Stool patterns

  Weeks 1, 4, 8, 12 and 16

• Satiety questionnaire

  Weeks 1, 4, 8, 12 and 16

Study Arms (3)

4 Weeks

EXPERIMENTAL

All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 4 weeks.

Dietary Supplement: PKU Sphere

8 Weeks

EXPERIMENTAL

All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 8 weeks.

Dietary Supplement: PKU Sphere

12 Weeks

EXPERIMENTAL

All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 12 weeks.

Dietary Supplement: PKU Sphere

Interventions

PKU Sphere DIETARY_SUPPLEMENT

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.

Also known as: GMP, Glycomacropeptide

12 Weeks; 4 Weeks; 8 Weeks

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Vitaflo International, Ltd: lead
• Kreiskliniken Reutlingen: collaborator
• Johannes Gutenberg University Mainz: collaborator

Study Sites (1)

Kreiskliniken Reutlingen

Reutlingen, Baden-Wurttemberg, D-72764, Germany

Location

MeSH Terms

Conditions

Phenylketonurias

Interventions

caseinomacropeptide

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Peter Freisinger

  Kreiskliniken Reutlingen GmbH

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Masking Details: At the point of randomisation every effort will be made to ensure that participants are unaware of the cluster they are randomised to; this is to help ensure that compliance is not affected by participants knowing when they will start the new treatment.
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The study has a stepped wedge design, which is chosen over the alternative cross-over study as it allows that all participants start on the amino acid supplement and avoids the difficulty of the 'wash-out' period being the same as one of the treatment strategies being offered.

Study Record Dates

• First Submitted: December 7, 2018
• First Posted: December 11, 2018
• Study Start: December 6, 2018
• Primary Completion: October 30, 2020
• Study Completion: July 31, 2023
• Last Updated: February 16, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)