Clinical trial NCT03771391: A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With 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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Phenylketonurias
Keywords: Phenylketonuria
PKU
Child
Children
Adult
Adults
GMP
Glycomacropeptide
Amino acid mixture
Amino acid
Protein supplement
Protein substitute
Lead sponsor: Vitaflo International, Ltd
Sponsor class: INDUSTRY
Collaborators: name: Kreiskliniken Reutlingen
name: Johannes Gutenberg University Mainz
First posted: 2018-12-11
Enrollment: 15
Sex: ALL
Minimum age (years): 10
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: 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.
Primary purpose: TREATMENT

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.

Detailed 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.

Eligibility 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

Primary outcomes:
measure: Change in blood Phe
timeFrame: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
Secondary outcomes:
measure: Change in blood tyrosine
timeFrame: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
measure: Dietary intake
timeFrame: Weeks 1 and 16.
measure: Protein substitute intake
timeFrame: Weeks 1, 4, 8, 12 and 16
measure: Stool patterns
timeFrame: Weeks 1, 4, 8, 12 and 16
measure: Satiety questionnaire
timeFrame: Weeks 1, 4, 8, 12 and 16
measure: Acceptance questionnaire
timeFrame: Weeks 1, 4, 8, 12 and 16
measure: Gastrointestinal symptoms questionnaire
timeFrame: Weeks 1, 4, 8, 12 and 16
Other outcomes:
Locations:
city: Reutlingen
country: Germany
facility: Kreiskliniken Reutlingen
state: Baden-Württemberg
Study officials:
affiliation: Kreiskliniken Reutlingen GmbH
name: Peter Freisinger
role: PRINCIPAL_INVESTIGATOR
References: