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GMP Drink for PKU Study

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ClinicalTrials.gov Identifier: NCT02915510
Recruitment Status : Completed
First Posted : September 27, 2016
Last Update Posted : October 1, 2019
Sponsor:
Information provided by (Responsible Party):
Nutricia UK Ltd

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

Condition or disease Intervention/treatment Phase
Phenylketonuria Dietary Supplement: Glycomacropeptide-based protein substitute Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official 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
Study Start Date : July 2016
Actual Primary Completion Date : September 27, 2019
Actual Study Completion Date : September 27, 2019


Arm Intervention/treatment
Experimental: GMP
Single arm designed, 3 day baseline, 28day on GMP
Dietary Supplement: Glycomacropeptide-based protein substitute



Primary Outcome Measures :
  1. Blood Phenylalanine (and other amino acids) [ Time Frame: 3 points throughout 28 days ]
    Blood spot test


Secondary Outcome Measures :
  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



Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02915510


Locations
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United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Guy's and St Thomas' NHS Fondation Trust
London, United Kingdom
UCLH
London, United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom
Sheffield Teaching Hospital
Sheffield, United Kingdom
Sponsors and Collaborators
Nutricia UK Ltd
Investigators
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Study Director: Rebecca Stratton, PHd Nutricia Ltd UK
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Responsible Party: Nutricia UK Ltd
ClinicalTrials.gov Identifier: NCT02915510    
Other Study ID Numbers: GMP2016
First Posted: September 27, 2016    Key Record Dates
Last Update Posted: October 1, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Phenylketonurias
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
Metabolic Diseases