PKU Low Calorie Drink Study

NCT04272736 | Withdrawn

• 1 other identifier: PKULite2018
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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).

Conditions

Phenylketonurias; Hyperphenylalaninaemia

Outcome Measures

Primary Outcomes (1)

• Compliance

  Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded

  Daily for 31 days

Secondary Outcomes (5)

• Acceptability: Tick-box questionnaire

  Recorded at baseline and end of intervetnion (day 31)

• Blood phenylalanine and other amino acid levels

  Recorded at baselien and end of intervention (day 31)

• Gastro-intestinal tolerance

  recorded at baseline, beginning of intervention and end of intervention(12 out of 31 days in total)

• Nutrient intake

  Recorded at basleine and end of intervention (day 31)

• Anthropometry

  Recorded at baseline and end of intervention (day 31)

Study Arms (1)

Low calorie protein substitute

EXPERIMENTAL

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

Dietary Supplement: Low calorie protein substitute

Interventions

Low calorie protein substitute DIETARY_SUPPLEMENT

The evaluated dietary supplement is a lower calorie amino acid based liquid protein substitute designed for patients with PKU.

Low calorie protein substitute

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Nutricia UK Ltd: lead

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 29, 2018
• First Posted: February 17, 2020
• Study Start: December 1, 2023
• Primary Completion: December 31, 2023
• Study Completion: December 31, 2023
• Last Updated: February 26, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share