GMP Drink for PKU Study
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
1 other identifier
interventional
40
1 country
5
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for not_applicable
Started Jul 2016
Typical duration for not_applicable
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
July 1, 2016
CompletedFirst Submitted
Initial submission to the registry
August 8, 2016
CompletedFirst Posted
Study publicly available on registry
September 27, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 27, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
September 27, 2019
CompletedOctober 1, 2019
September 1, 2019
3.2 years
August 8, 2016
September 27, 2019
Conditions
Outcome Measures
Primary Outcomes (1)
Blood Phenylalanine (and other amino acids)
Blood spot test
3 points throughout 28 days
Secondary Outcomes (7)
Gastro-intestinal tolerance questionaire
12/31 days
Product compliance questionaire
31 days
Product acceptability questionnaire
2/31 days
Patient reproted nutrient intake (3 day food diaries)
6/31 days
Pateint-reported appetite (visual analogue scale)
6/31 days
- +2 more secondary outcomes
Study Arms (1)
GMP
EXPERIMENTALSingle arm designed, 3 day baseline, 28day on GMP
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Nutricia UK Ltdlead
Study Sites (5)
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
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Rebecca Stratton, PHd
Nutricia Ltd UK
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 8, 2016
First Posted
September 27, 2016
Study Start
July 1, 2016
Primary Completion
September 27, 2019
Study Completion
September 27, 2019
Last Updated
October 1, 2019
Record last verified: 2019-09
Data Sharing
- IPD Sharing
- Will not share