GMP Drink for PKU Study

Nutricia

Status: Completed

Conditions: Phenylketonuria

Treatments: Dietary Supplement: Glycomacropeptide-based protein substitute

Study type: Interventional

Funder types: Industry

Identifiers

NCT02915510

GMP2016

Details and patient eligibility

About

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

Enrollment: 40 patients

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Supportive Care

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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40 participants in 1 patient group

GMP

Experimental group

Description:

Single arm designed, 3 day baseline, 28day on GMP

Treatment:

Dietary Supplement: Glycomacropeptide-based protein substitute