Market Research - Acceptability Trial for a New PKU Amino... | NCT04309331

Studies
Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute

NCT04309331

Sponsor

Dr. Schär AG / SPA

Status

Completed

Last Update Posted

Mar 16, 2020Actual

Enrollment

12Actual

Phase

Not Applicable

Conditions

PKU
Phenylketonurias
Inborn Errors of Metabolism
Metabolic Disease

Interventions

Motion

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

Mar 2020

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 1, 2018Actual

Primary Completion Date

Sep 19, 2018Actual

Completion Date

Sep 19, 2018Actual

First Submitted Date

Feb 13, 2020

First Submission Date that Met QC Criteria

Mar 12, 2020

First Posted Date

Mar 16, 2020Actual

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

Mar 12, 2020

Last Update Posted Date

Mar 16, 2020Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

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

The aim of this study is to demonstrate that a new protein substitute is acceptable and well tolerated in children with 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

12Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
PKU Motion	Experimental	amino acid based protein substitute	Dietary Supplement: Motion

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Motion	Dietary Supplement	Amino acid based protein substitute	PKU Motion

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Gastrointestinal tolerance	Questionnaire detailing any GI symptoms, severity and change from usual • The collection of daily data about the gastro-intestinal tolerance of the protein substitute.	7 days
Product compliance	Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.	7 days
Product palatability	Questionnaire data captured to evaluate taste	7 days
Product acceptability	Brief tick-box questionnaire on overall liking and acceptability of product	7 days

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Diagnosis of PKU or PKU variant requiring a phenylalanine-free protein substitute
Subjects who are already taking a phenylalanine-free protein substitute and are willing to try the study product for 7 days
Patients aged 3 years of age to adulthood
Written informed consent obtained from parental caregiver

Exclusion Criteria:

Presence of serious concurrent illness
Investigator's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
Any patients having taken antibiotics over the previous 2 weeks leading up to the study.
Patients less than 3 years of age.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

3 Years

Maximum Age

16 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Birmingham Children's Hospital		Birmingham		B4 6NH	United Kingdom

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
D008661	Metabolism, Inborn Errors
D008659	Metabolic Diseases

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