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Completed

NCT Number: NCT02915510

GMP Drink for PKU Study

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

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Key information

Conditions

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

Age range

3 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Great Ormond Street Hospital, London, United Kingdom

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Glycomacropeptide-based protein substitute

Dietary Supplement

Primary outcomes

  1. Blood Phenylalanine (and other amino acids)

    Time frame: 3 points throughout 28 days

    Blood spot test

Secondary outcomes

  1. Gastro-intestinal tolerance questionaire

    Time frame: 12/31 days

    Questionaire detailing any GI symptoms, severity and change from usual

  2. Product compliance questionaire

    Time frame: 31 days

    Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount.

  3. Product acceptability questionnaire

    Time frame: 2/31 days

    Breif tick-box questionaire on overal liking and acceptability of product

  4. Patient reproted nutrient intake (3 day food diaries)

    Time frame: 6/31 days

    3 day food diaries, subsequently analysed in dietary software.

  5. Pateint-reported appetite (visual analogue scale)

    Time frame: 6/31 days

    Measures of overal appetite and satiety via visual analouge scale

  6. Antropometry (hieight and weight)

    Time frame: 2/31 days

    Measurements of height and weight at baseline and end of study

  7. Safety (Adverse events reporting)

    Time frame: 31 days

    Reporting of adverse events throughout study

Sponsors and collaborators

Lead sponsor

Nutricia UK Ltd

Industry

Registry information

Official study 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

Important dates

Study start
2016
Primary completion
2019
Study completion
2019
First posted
Sep 27, 2016
Registry last updated
Oct 1, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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