GMP Drink for PKU Study

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02915510
• Date of registration: 08/08/2016
• Prospective Registration: No
• Primary sponsor: Nutricia UK Ltd
• Public title: GMP Drink for PKU Study
• Scientific 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
• Date of first enrolment: July 2016
• Target sample size: 40
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02915510
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Supportive Care. Masking: None (Open Label).
• Phase: N/A

Countries of recruitment

United Kingdom

Contacts

• Name: Rebecca Stratton, PHd
• Affiliation: Nutricia Ltd UK

Key inclusion & exclusion criteria

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



Age minimum: 3 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Phenylketonuria

Intervention(s)

Dietary Supplement: Glycomacropeptide-based protein substitute

Primary Outcome(s)

Blood Phenylalanine (and other amino acids) [Time Frame: 3 points throughout 28 days]

Secondary Outcome(s)

Patient reproted nutrient intake (3 day food diaries) [Time Frame: 6/31 days]

Safety (Adverse events reporting) [Time Frame: 31 days]

Pateint-reported appetite (visual analogue scale) [Time Frame: 6/31 days]

Product compliance questionaire [Time Frame: 31 days]

Antropometry (hieight and weight) [Time Frame: 2/31 days]

Gastro-intestinal tolerance questionaire [Time Frame: 12/31 days]

Product acceptability questionnaire [Time Frame: 2/31 days]

Secondary ID(s)

GMP2016

Source(s) of Monetary Support

Please refer to primary and secondary sponsors