GMP Drink for PKU Study | NCT02915510

Studies
GMP Drink for PKU Study

NCT02915510

Sponsor

Nutricia UK Ltd

Status

Completed

Last Update Posted

Oct 1, 2019Actual

Enrollment

40Actual

Phase

Not Applicable

Conditions

Phenylketonuria

Interventions

Glycomacropeptide-based protein substitute

Countries

United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Sep 2019

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 2016

Primary Completion Date

Sep 27, 2019Actual

Completion Date

Sep 27, 2019Actual

First Submitted Date

Aug 8, 2016

First Submission Date that Met QC Criteria

Sep 22, 2016

First Posted Date

Sep 27, 2016Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 27, 2019

Last Update Posted Date

Oct 1, 2019Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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).

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

40Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
GMP	Experimental	Single arm designed, 3 day baseline, 28day on GMP	Dietary Supplement: Glycomacropeptide-based protein substitute

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Glycomacropeptide-based protein substitute	Dietary Supplement		GMP

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Measure	Description	Time Frame
Gastro-intestinal tolerance questionaire	Questionaire detailing any GI symptoms, severity and change from usual	12/31 days
Product compliance questionaire	Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount.

Other Outcomes

Not provided

Eligibility Module

Eligibility 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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

3 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Rebecca Stratton, PHd	Nutricia Ltd UK	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Great Ormond Street Hospital		London			United Kingdom
Guy's and St Thomas' NHS Fondation Trust

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D010661	Phenylketonurias

Ancestor Terms

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided