GMP Drink for PKU Study

Not Applicable

Completed

• Conditions: Phenylketonuria
• Interventions: Dietary Supplement: Glycomacropeptide-based protein substitute

• Registration Number: NCT02915510
• Lead Sponsor: Nutricia UK Ltd

Brief Summary

This study will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU).

Detailed Description

Not available

Study Timeline

• Study Start: 2016-07
• Study First Submitted: 2016-08-08
• Study First Submitted QC: 2016-09-22
• Study First Posted: 2016-09-27
• Status Verified: 2019-09
• Primary Completion: 2019-09-27
• Study Completion: 2019-09-27
• Last Update Submitted: 2019-09-27
• Last Update Posted: 2019-10-01

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Male or female
• Over 3 years of age
• Diagnosed with classical or variant type phenylketonuria
• Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalents, for at least 1 month prior to trial commencement
• Have a prescribed daily Phe allowance
• Written informed consent from patient, or from parent / carer if applicable

Exclusion Criteria

• Currently prescribed Sapropterin or similar tetrahydrobiopterin based medication
• Pregnant or lactating
• Requiring parenteral nutrition
• Major hepatic or renal dysfunction
• Participation in other studies within 1 month prior to entry of this study
• Allergy to any of the study product ingredients, including milk protein or soya
• Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
GMP	Glycomacropeptide-based protein substitute	Single arm designed, 3 day baseline, 28day on GMP

Primary Outcome Measures

Name	Time	Method
Blood Phenylalanine (and other amino acids)	3 points throughout 28 days	Blood spot test

Secondary Outcome Measures

Name	Time	Method
Gastro-intestinal tolerance questionaire	12/31 days	Questionaire detailing any GI symptoms, severity and change from usual
Product compliance questionaire	31 days	Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount.
Product acceptability questionnaire	2/31 days	Breif tick-box questionaire on overal liking and acceptability of product
Patient reproted nutrient intake (3 day food diaries)	6/31 days	3 day food diaries, subsequently analysed in dietary software.
Pateint-reported appetite (visual analogue scale)	6/31 days	Measures of overal appetite and satiety via visual analouge scale
Antropometry (hieight and weight)	2/31 days	Measurements of height and weight at baseline and end of study
Safety (Adverse events reporting)	31 days	Reporting of adverse events throughout study

Trial Locations

Locations (5)

Great Ormond Street Hospital; 🇬🇧 London, United Kingdom

Royal Manchester Children's Hospital; 🇬🇧 Manchester, United Kingdom

UCLH; 🇬🇧 London, United Kingdom

Guy's and St Thomas' NHS Fondation Trust; 🇬🇧 London, United Kingdom

Sheffield Teaching Hospital; 🇬🇧 Sheffield, United Kingdom