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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-12-06

Study Completion Date

2023-07-31

Brief Summary

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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.

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Detailed Description

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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.

Conditions

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Phenylketonurias

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

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 Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Participants

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.

Study Groups

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4 Weeks

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.

Group Type EXPERIMENTAL

PKU Sphere

Intervention Type 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.

8 Weeks

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.

Group Type EXPERIMENTAL

PKU Sphere

Intervention Type 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.

12 Weeks

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.

Group Type EXPERIMENTAL

PKU Sphere

Intervention Type 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.

Interventions

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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.

Intervention Type DIETARY_SUPPLEMENT

Other Intervention Names

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GMP Glycomacropeptide

Eligibility Criteria

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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

Minimum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No