GMP Drink for PKU Study

Phenylketonuria Clinical Trial

Official title:

Evaluating the Tolerance, Safety and Acceptability of PKU GMPro, a Whey Protein Derived Feed for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02915510
• Other study ID #: GMP2016
• Status: Completed
• Phase: N/A
• Start date: July 2016
• Est. completion date: September 27, 2019

Study information

• Verified date: September 2019
• Source: Nutricia UK Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: September 27, 2019
• Est. primary completion date: September 27, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Phenylketonuria
• Phenylketonurias

Intervention

Dietary Supplement:
• Glycomacropeptide-based protein substitute

Locations

Country	Name	City	State
United Kingdom	Great Ormond Street Hospital	London
United Kingdom	Guy's and St Thomas' NHS Fondation Trust	London
United Kingdom	UCLH	London
United Kingdom	Royal Manchester Children's Hospital	Manchester
United Kingdom	Sheffield Teaching Hospital	Sheffield

Sponsors (1)

Lead Sponsor	Collaborator
Nutricia UK Ltd

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Blood Phenylalanine (and other amino acids)	Blood spot test	3 points throughout 28 days
Secondary	Gastro-intestinal tolerance questionaire	Questionaire detailing any GI symptoms, severity and change from usual	12/31 days
Secondary	Product compliance questionaire	Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount.	31 days
Secondary	Product acceptability questionnaire	Breif tick-box questionaire on overal liking and acceptability of product	2/31 days
Secondary	Patient reproted nutrient intake (3 day food diaries)	3 day food diaries, subsequently analysed in dietary software.	6/31 days
Secondary	Pateint-reported appetite (visual analogue scale)	Measures of overal appetite and satiety via visual analouge scale	6/31 days
Secondary	Antropometry (hieight and weight)	Measurements of height and weight at baseline and end of study	2/31 days
Secondary	Safety (Adverse events reporting)	Reporting of adverse events throughout study	31 days