GMP Drink for PKU Study

September 27, 2019 updated by: Nutricia UK Ltd

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

40

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • London, United Kingdom
        • Great Ormond Street Hospital
      • London, United Kingdom
        • UCLH
      • London, United Kingdom
        • Guy's and St Thomas' NHS Fondation Trust
      • Manchester, United Kingdom
        • Royal Manchester Children's Hospital
      • Sheffield, United Kingdom
        • Sheffield Teaching Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Supportive Care
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: GMP
Single arm designed, 3 day baseline, 28day on GMP
Dietary supplement: Glycomacropeptide-based protein substitute

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Blood Phenylalanine (and other amino acids)
Time Frame: 3 points throughout 28 days
Blood spot test
3 points throughout 28 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Gastro-intestinal tolerance questionaire
Time Frame: 12/31 days
Questionaire detailing any GI symptoms, severity and change from usual
12/31 days
Product compliance questionaire
Time Frame: 31 days
Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount.
31 days
Product acceptability questionnaire
Time Frame: 2/31 days
Breif tick-box questionaire on overal liking and acceptability of product
2/31 days
Patient reproted nutrient intake (3 day food diaries)
Time Frame: 6/31 days
3 day food diaries, subsequently analysed in dietary software.
6/31 days
Pateint-reported appetite (visual analogue scale)
Time Frame: 6/31 days
Measures of overal appetite and satiety via visual analouge scale
6/31 days
Antropometry (hieight and weight)
Time Frame: 2/31 days
Measurements of height and weight at baseline and end of study
2/31 days
Safety (Adverse events reporting)
Time Frame: 31 days
Reporting of adverse events throughout study
31 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nutricia UK Ltd

Investigators

  • Study Director: Rebecca Stratton, PHd, Nutricia Ltd UK

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2016

Primary Completion (Actual)

September 27, 2019

Study Completion (Actual)

September 27, 2019

Study Registration Dates

First Submitted

August 8, 2016

First Submitted That Met QC Criteria

September 22, 2016

First Posted (Estimate)

September 27, 2016

Study Record Updates

Last Update Posted (Actual)

October 1, 2019

Last Update Submitted That Met QC Criteria

September 27, 2019

Last Verified

September 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GMP2016

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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