PKU Low Calorie Drink Study

Nutricia

Status: Withdrawn

Conditions: Phenylketonurias; Hyperphenylalaninaemia

Treatments: Dietary Supplement: Low calorie protein substitute

Study type: Interventional

Funder types: Industry

Identifiers

NCT04272736

PKULite2018

Details and patient eligibility

About

This study will evaluate the compliance, acceptability, gastrointestinal (GI) tolerance and safety of a lower calorie amino acid based liquid protein substitute for patients with Phenylketonuria (PKU) or hyperphenylalaninemia (HPA).

Sex: All

Ages: 3+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

• Pregnant or lactating
• Requiring enteral tube or parenteral nutrition
• Major hepatic or renal dysfunction
• Participation in other studies within 1 month prior to entry to this study
• Allergy to any of the study product ingredients
• Investigator concern around the patient being underweight (e.g.: BMI lower than 18.5kg/m2 for adults) and/or having an eating disorder
• 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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0 participants in 1 patient group

Low calorie protein substitute

Experimental group

Description:

Single arm designed, 3 day baseline, 28 day on the lower calorie amino acid based liquid protein substitute.

Treatment:

Dietary Supplement: Low calorie protein substitute